I am from Columbus, Ohio. I was diagnosed with PH 2009, but that's not where my journey started. I was born with a hole in my heart, back in 1956. So not a lot of treatment, not a lot of surgeries that went well. I was one of the lucky ones. They were able to do open heart surgery, put a patch on it. So I went through my life not being able to take gym or doing a lot of stuff like that. I was told not to have any kids, to really watch myself.

Well, I ended up with three daughters, no problems. Relatively good health, until I was 40. They found out that I had a tear in my aortic valve, so I had a second open heart surgery. For 40 years, I was not on any medication. When I hit 40 is when I started that. Then my health went in peaks, off and on, until I was diagnosed when I was 54 with pulmonary hypertension. I went in not being able to walk very far, very out of breath. Couldn't do stairs. I went to my regular doctor thinking, "Okay, I'm just getting older, I'm overweight, just like everybody thought." Or, back in my mind, maybe it was my heart.

My doctor had me walk around the hall, and my oxygen was 67 at that time. She said, "Joellen, I can't send you home without oxygen, and you can't drive home." So that was my day with oxygen. I had to have my daughter come and get me. But back then, I didn't know what was happening, so she sent me to a cardiologist that specializes in PH. And that's when I had my first right heart catheterization, and he diagnosed me then.

For me, I know when you're first diagnosed with PH, it's very scary. For me, for 54 years, I've had this heart condition and to me, it was just one other thing. At the time, I didn't know how serious it was, so I'm thinking it's just one other thing I'm going to have to deal with in my life, and I'll just go to the doctor and he's just going to say again, "Lose weight, get in better shape, and you'll be fine." So when you are with that diagnosis, yes you go home and you look it up on the internet, which is not a real good thing to do. It was just like, "Okay. Now what am I going to do?"

So the two things for me was being on oxygen, and the other one was having to quit my job. Those two things, because at 54, you're thinking you're going to be able to stay at this job until you retire. I worked at a call center, so I thought, "Well this is a good job for me. I'm sitting down, I'm not doing anything strenuous." But when you're on the phone eight hours a day, you get out of breath really easy. And I was trying to carry those little portable tanks with me, and I had to walk out in the boonies. It was just getting too much.

Getting into a support group meeting, where you meet (other patients), that has been my saving grace. That helps me, going one on one, meeting people, getting to be friends with them, hearing their stories. My support group is small, and so a lot of us have the same PH doctor, so we're able to relate all with that, so that's been good.

Make sure you get a support group in your area, because you're really going to need that, because family and friends are not going to understand. Even though they say they want to help, they're not going to understand what it feels like getting up in the morning and it's like, "I don't want to get out of bed. I just don't want to." I would say those things is what you really need to do.

I've heard of a lot of people say, "Some of my family don't even know that I have this." My girls, I told them right off. I explained to them what it was, so I've kept them in the loop about everything. Maybe I'm not supposed to, maybe I tell them too much. So they understand. They're getting the awareness out there, it's like little fingers that just trinkle on there.

Even with my heart condition, I was always doing research studies at my hospital. It's a university, so it's a teaching hospital. I was always like a guinea pig. I always feel like if I can help out, so I do the phone studies. They'll call you and you do things like this. I can do that, I can do that over the phone, and I can answer questions, and I can talk about new treatments coming up and all that, so I try to do that part of it. I'm not physical enough to do the marathons and the walks and all that stuff, so I feel like if I can do those little surveys, then that's my thing that I can do.

]]>In this episode, pulmonary hypertension patient Joellen Brown discusses being born with a hole in her heart in 1956 that developed into a PH diagnosis in 2009 and the importance of support groups and PH research.

My name is Joellen Brown, and I am a pulmonary hypertension patient.

I am from Columbus, Ohio. I was diagnosed with PH 2009, but that's not where my journey started. I was born with a hole in my heart, back in 1956. So not a lot of treatment, not a lot of surgeries that went well. I was one of the lucky ones. They were able to do open heart surgery, put a patch on it. So I went through my life not being able to take gym or doing a lot of stuff like that. I was told not to have any kids, to really watch myself.

Well, I ended up with three daughters, no problems. Relatively good health, until I was 40. They found out that I had a tear in my aortic valve, so I had a second open heart surgery. For 40 years, I was not on any medication. When I hit 40 is when I started that. Then my health went in peaks, off and on, until I was diagnosed when I was 54 with pulmonary hypertension. I went in not being able to walk very far, very out of breath. Couldn't do stairs. I went to my regular doctor thinking, "Okay, I'm just getting older, I'm overweight, just like everybody thought." Or, back in my mind, maybe it was my heart.

My doctor had me walk around the hall, and my oxygen was 67 at that time. She said, "Joellen, I can't send you home without oxygen, and you can't drive home." So that was my day with oxygen. I had to have my daughter come and get me. But back then, I didn't know what was happening, so she sent me to a cardiologist that specializes in PH. And that's when I had my first right heart catheterization, and he diagnosed me then.

For me, I know when you're first diagnosed with PH, it's very scary. For me, for 54 years, I've had this heart condition and to me, it was just one other thing. At the time, I didn't know how serious it was, so I'm thinking it's just one other thing I'm going to have to deal with in my life, and I'll just go to the doctor and he's just going to say again, "Lose weight, get in better shape, and you'll be fine." So when you are with that diagnosis, yes you go home and you look it up on the internet, which is not a real good thing to do. It was just like, "Okay. Now what am I going to do?"

So the two things for me was being on oxygen, and the other one was having to quit my job. Those two things, because at 54, you're thinking you're going to be able to stay at this job until you retire. I worked at a call center, so I thought, "Well this is a good job for me. I'm sitting down, I'm not doing anything strenuous." But when you're on the phone eight hours a day, you get out of breath really easy. And I was trying to carry those little portable tanks with me, and I had to walk out in the boonies. It was just getting too much.

Getting into a support group meeting, where you meet (other patients), that has been my saving grace. That helps me, going one on one, meeting people, getting to be friends with them, hearing their stories. My support group is small, and so a lot of us have the same PH doctor, so we're able to relate all with that, so that's been good.

Make sure you get a support group in your area, because you're really going to need that, because family and friends are not going to understand. Even though they say they want to help, they're not going to understand what it feels like getting up in the morning and it's like, "I don't want to get out of bed. I just don't want to." I would say those things is what you really need to do.

I've heard of a lot of people say, "Some of my family don't even know that I have this." My girls, I told them right off. I explained to them what it was, so I've kept them in the loop about everything. Maybe I'm not supposed to, maybe I tell them too much. So they understand. They're getting the awareness out there, it's like little fingers that just trinkle on there.

Even with my heart condition, I was always doing research studies at my hospital. It's a university, so it's a teaching hospital. I was always like a guinea pig. I always feel like if I can help out, so I do the phone studies. They'll call you and you do things like this. I can do that, I can do that over the phone, and I can answer questions, and I can talk about new treatments coming up and all that, so I try to do that part of it. I'm not physical enough to do the marathons and the walks and all that stuff, so I feel like if I can do those little surveys, then that's my thing that I can do.

]]>05:43cleanblood,research,heart,awareness,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseaseIn this episode, pulmonary hypertension patient Joellen Brown discusses being born with a hole in her heart in 1956 that developed into a PH diagnosis in 2009 and the importance of support groups and PH research.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials @antidote_me332fullphaware global associationHollie Satona - phaware® interview 230Tue, 19 Mar 2019 12:00:00 +0000

Pediatric pulmonary hypertension caregiver, Hollie Satona discusses her son Joelan's PH diagnosis and the challenge of juggling caregiving because three of her four children are impacted by rare disease and special needs.

I'm Hollie Satona and I am the parent and caregiver of my son who is eight years old, Joelan, who has severe pulmonary hypertension.

It's been kind of a whirlwind for JoJo since the day he was born. I didn't know about pulmonary hypertension until about two years ago. I kind of wish I would have known some of the symptoms prior to diagnosis, because I think he could have been diagnosed sooner. He was born with a large VSD and ASD and those were both repaired when he was six months old. He seemed to be a normal kid after those repaired procedures, until he was six years old and decided he wanted to go to T-ball.

At T-ball practice, the first night, he couldn't keep up. His heart was pounding. You could visibly see his murmur, visibly see it. So I'm like something is not right here. I thought maybe one of the holes came back or there is something minor going on. We went to the doctor and he told me I was crazy. He's like oh, he's just overtired from running bases in T-ball. So then the next day, I took him to his family doctor and I had him run up and down the hall and as soon as he stopped and the doctor looked at him, he could see exactly what I was talking about. He was instantly on the phone with the cardiologist. It's been a whirlwind since then.

Once we figured out getting him to the right doctors, the diagnosis was pretty quick. It was the first echocardiogram, the first cardiologist appointment after that we were sent to UW Madison in Wisconsin to do a right-heart catheterization. June 28, 2016, was his confirmed diagnosis. His pressures were over 145 at the catheterization. We got a rough start, a very quick informed theory of what pulmonary hypertension was and the therapies.

It has been a challenge. The one thing I feel, though, is I kind of fight through challenges a lot with our lives. Our families are pretty busy. It was an obstacle that overcomes a busy lifestyle, because pulmonary hypertension definitely puts the brakes on a lot of obstacles that we face. Throughout the diagnosis, there's been quite a few hospital stays and procedures that he's had done and some evaluations just because of the severity of his pressures.

Right now, he's doing wonderful. We finally got everything controlled, but we definitely learned to juggle. It's like a circus, so we definitely are good at juggling between the kids because there are four boys in the family. There is a lot of consideration. We have a lot of good moral support and help with everything in the house. I'm pretty lucky on that side.

Not only does JoJo have pulmonary hypertension, but his older brother has hydrocephalus and epilepsy, and his younger brother has a cognitive disorder and he is on hearing aids. So, between the three out of the four boys, we've been familiar with multitasking for a long time between all of the doctors we see and specialists.

My sister has been really a good support with the caregiver side of things, as well, so she's trained. My oldest son, who's 14, is actually trained to mix the medication and stuff for Joelan. We try to balance everything out. His dad has been more of a moral support because he's not as strong, I think, as myself where, you know, sometimes that scares people. It's too much for some people and it really is. There's a lot that plays into consideration. But the challenges between work, I work full time, as well, as an accountant. And my job and my bosses have been very considerate of the situation. If I have to leave work, there's no question. I can literally drop and go. I work two miles from my house, so it literally takes me two minutes to get to the school, two minutes to get home. The doctor's office, not so much. It's about a two-hour drive.

We've got a lot of responses in place in case of situations, whether it's Airlink ambulance. I am fortunate that one of the first responders is on my staff, so she's literally two steps away from me. So she gets the first call and then she's like, oh, let's go, Holly. But we haven't had that. We've got a lot of structures in place to consider in case something goes wrong.

When JoJo was referred to St. Louis for a transplant evaluation, we were informed about the Pott shunt procedure. That's scary because for pediatrics, it's only been done, at that time, maybe only 12 times in the United States. And his cardiologist, the one thing she told me, she was like, "That's kind of a death wish. Would you really risk your son's life?" And then, for me, I answered, "Isn't he already got one? You just told me he has pulmonary hypertension. You said most chances if he lives over five years." So you feel like you're backed in a corner. So, for me personally, to overcome these obstacles there's two options you have when you're pushed in a corner. You can sit there, cry about it, and do nothing. Or you can push forward and fight for it. And that's definitely how I do and how I handle things.

I've overcome a lot of challenges aside from pulmonary hypertension, but it's definitely made it stronger to move forward with the advances in the medication and therapies, to put trust in someone else's hands for the care of my son.

Do the research. There's a lot of research that is available to do on your own. Don't be afraid to ask questions and definitely don't be afraid to challenge them. There's something much stuff that's coming out new that you may see or hear about and they might not be familiar with those. Always push through and make sure the doctors are allowing you to have that responsibility and respect. I'm really good at double checking everything and that's the one thing you have to do. You protect your child. So questions, never be afraid to ask questions.

Pediatric pulmonary hypertension caregiver, Hollie Satona discusses her son Joelan's PH diagnosis and the challenge of juggling caregiving because three of her four children are impacted by rare disease and special needs.

I'm Hollie Satona and I am the parent and caregiver of my son who is eight years old, Joelan, who has severe pulmonary hypertension.

It's been kind of a whirlwind for JoJo since the day he was born. I didn't know about pulmonary hypertension until about two years ago. I kind of wish I would have known some of the symptoms prior to diagnosis, because I think he could have been diagnosed sooner. He was born with a large VSD and ASD and those were both repaired when he was six months old. He seemed to be a normal kid after those repaired procedures, until he was six years old and decided he wanted to go to T-ball.

At T-ball practice, the first night, he couldn't keep up. His heart was pounding. You could visibly see his murmur, visibly see it. So I'm like something is not right here. I thought maybe one of the holes came back or there is something minor going on. We went to the doctor and he told me I was crazy. He's like oh, he's just overtired from running bases in T-ball. So then the next day, I took him to his family doctor and I had him run up and down the hall and as soon as he stopped and the doctor looked at him, he could see exactly what I was talking about. He was instantly on the phone with the cardiologist. It's been a whirlwind since then.

Once we figured out getting him to the right doctors, the diagnosis was pretty quick. It was the first echocardiogram, the first cardiologist appointment after that we were sent to UW Madison in Wisconsin to do a right-heart catheterization. June 28, 2016, was his confirmed diagnosis. His pressures were over 145 at the catheterization. We got a rough start, a very quick informed theory of what pulmonary hypertension was and the therapies.

It has been a challenge. The one thing I feel, though, is I kind of fight through challenges a lot with our lives. Our families are pretty busy. It was an obstacle that overcomes a busy lifestyle, because pulmonary hypertension definitely puts the brakes on a lot of obstacles that we face. Throughout the diagnosis, there's been quite a few hospital stays and procedures that he's had done and some evaluations just because of the severity of his pressures.

Right now, he's doing wonderful. We finally got everything controlled, but we definitely learned to juggle. It's like a circus, so we definitely are good at juggling between the kids because there are four boys in the family. There is a lot of consideration. We have a lot of good moral support and help with everything in the house. I'm pretty lucky on that side.

Not only does JoJo have pulmonary hypertension, but his older brother has hydrocephalus and epilepsy, and his younger brother has a cognitive disorder and he is on hearing aids. So, between the three out of the four boys, we've been familiar with multitasking for a long time between all of the doctors we see and specialists.

My sister has been really a good support with the caregiver side of things, as well, so she's trained. My oldest son, who's 14, is actually trained to mix the medication and stuff for Joelan. We try to balance everything out. His dad has been more of a moral support because he's not as strong, I think, as myself where, you know, sometimes that scares people. It's too much for some people and it really is. There's a lot that plays into consideration. But the challenges between work, I work full time, as well, as an accountant. And my job and my bosses have been very considerate of the situation. If I have to leave work, there's no question. I can literally drop and go. I work two miles from my house, so it literally takes me two minutes to get to the school, two minutes to get home. The doctor's office, not so much. It's about a two-hour drive.

We've got a lot of responses in place in case of situations, whether it's Airlink ambulance. I am fortunate that one of the first responders is on my staff, so she's literally two steps away from me. So she gets the first call and then she's like, oh, let's go, Holly. But we haven't had that. We've got a lot of structures in place to consider in case something goes wrong.

When JoJo was referred to St. Louis for a transplant evaluation, we were informed about the Pott shunt procedure. That's scary because for pediatrics, it's only been done, at that time, maybe only 12 times in the United States. And his cardiologist, the one thing she told me, she was like, "That's kind of a death wish. Would you really risk your son's life?" And then, for me, I answered, "Isn't he already got one? You just told me he has pulmonary hypertension. You said most chances if he lives over five years." So you feel like you're backed in a corner. So, for me personally, to overcome these obstacles there's two options you have when you're pushed in a corner. You can sit there, cry about it, and do nothing. Or you can push forward and fight for it. And that's definitely how I do and how I handle things.

I've overcome a lot of challenges aside from pulmonary hypertension, but it's definitely made it stronger to move forward with the advances in the medication and therapies, to put trust in someone else's hands for the care of my son.

Do the research. There's a lot of research that is available to do on your own. Don't be afraid to ask questions and definitely don't be afraid to challenge them. There's something much stuff that's coming out new that you may see or hear about and they might not be familiar with those. Always push through and make sure the doctors are allowing you to have that responsibility and respect. I'm really good at double checking everything and that's the one thing you have to do. You protect your child. So questions, never be afraid to ask questions.

]]>fullEpisode 230 - Hollie SatonaHollie Satona - Pulmonary HypertensionMon, 18 Mar 2019 12:00:00 +0000In this episode, pediatric pulmonary hypertension caregiver, Hollie Satona discusses her son Joelan's PH diagnosis and the challenge of juggling caregiving because three of her four children are impacted by rare disease and special needs.

]]>In this episode, pediatric pulmonary hypertension caregiver, Hollie Satona discusses her son Joelan's PH diagnosis and the challenge of juggling caregiving because three of her four children are impacted by rare disease and special needs.

]]>05:50cleanblood,research,heart,pediatric,awareness,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseaseIn this episode, pediatric pulmonary hypertension caregiver, Hollie Satona discusses her son Joelan's PH diagnosis and the challenge of juggling caregiving because three of her four children are impacted by rare disease and special needs.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware. Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials @antidote_me331fullphaware global associationBarbara Thompson - phaware® interview 229Fri, 15 Mar 2019 12:00:00 +0000

Pulmonary hypertension patient Barbara Thompson discusses why she wears zebra clothing 365 days a year and her long road through 14 doctors before a young grad student gave her a proper PH diagnosis.

Hi, I'm Barbara Thompson. I'm a pulmonary hypertension patient.

We used to do weddings as a family business. We did everything: photography, video, cakes, catering, flowers, directing, reception hall, chapel. But the last thing I remember in April of 2000, was getting a bride down the aisle, and then I don't remember anything until December of 2002, because when your brain is not getting enough oxygen, you don't remember things. I became bedridden.

My husband, Chuck, took me to 14 different doctors before I was diagnosed. 14th doctor had just graduated from medical school, so he was a little gung ho with stuff. And thank God he was. He said, “nobody has bronchitis this long. Let's look and let's take an echocardiogram, see what's going on.” So, he did that, and he called back in about week, and he said, “you probably need to come into the office and let's talk about this.” I said well what's going on, and he said, “pulmonary hypertension.” And all I heard was hypertension. And I said okay, hey give me a pill, and we're ready to go. And he said, “it's a little bit more complicated than that.” Then I had right heart catheterization and even though my numbers were very low at 27, 31, I was class 4. They said for our daughter that was getting married very soon, to move her wedding date up if they wanted me to be there. They basically gave me 6 to 9 months to survive and December 12, 2018, it'll be 16 years.

It's been very frustrating, because people aren't aware of the disease. I wear a zebra every day to help raise awareness. I developed a little card that explains the (medical) zebra analogy on one side, and a little bit about PH or pulmonary hypertension on the other side. I give those out on a regular basis. I've probably given out five or six thousand of them over the years. That's the purpose of wearing zebra every day, 365 days a year. When people look at me funny, I'll take a card out of my pocket, pants always have to have pockets, whip out a card, give it to them and say this is why I wear zebra. Just take a look at it, this will explain the zebra analogy to you a little bit and pulmonary hypertension on the other side. Then I'm on my way again, so they don't feel like they're compromised. And I'll tell cashiers if they say something about the oxygen. I'll give them a card and just say well, read this when you get a chance.

My mother-in-law died from pulmonary hypertension, as did my sister-in-law and probably a grandfather-in-law. So, it's familial on my husband's side. And then it's idiopathic on my side. People need to be aware of this, because my sister-in-law went to bed one night and didn't wake up the next morning and she was 35 years old. At the autopsy is where they saw that there was a problem with pulmonary artery. It had constricted. And they said that was the reason for her passing.

If you don't understand something ask. If you don't understand that, get on the Facebook groups. There's always somebody on Facebook that can answer your question. I have a list of places that will explain pulmonary hypertension in lay terms, as well as scientific terms or medical terms, that I send those links to different people, so they can understand what's going on with their body.

One thing that I wish I had known that a doctor didn't tell me was that this is not a death sentence. There is hope. Try to stay upbeat. Wear zebra. My mission in all of this is that when a woman feels a lump in her breast, she goes to her doctor and says could this be breast cancer. I want when a person has shortness of breath, fatigue, bluish tint to their lips, nail beds, swollen ankles, the extreme fatigue, which was my worst thing, and also hoarseness, because of the enlarged pulmonary artery. When they have those symptoms I want them to go to the doctor and say could this be pulmonary hypertension?

Pulmonary hypertension patient Barbara Thompson discusses why she wears zebra clothing 365 days a year and her long road through 14 doctors before a young grad student gave her a proper PH diagnosis.

Hi, I'm Barbara Thompson. I'm a pulmonary hypertension patient.

We used to do weddings as a family business. We did everything: photography, video, cakes, catering, flowers, directing, reception hall, chapel. But the last thing I remember in April of 2000, was getting a bride down the aisle, and then I don't remember anything until December of 2002, because when your brain is not getting enough oxygen, you don't remember things. I became bedridden.

My husband, Chuck, took me to 14 different doctors before I was diagnosed. 14th doctor had just graduated from medical school, so he was a little gung ho with stuff. And thank God he was. He said, “nobody has bronchitis this long. Let's look and let's take an echocardiogram, see what's going on.” So, he did that, and he called back in about week, and he said, “you probably need to come into the office and let's talk about this.” I said well what's going on, and he said, “pulmonary hypertension.” And all I heard was hypertension. And I said okay, hey give me a pill, and we're ready to go. And he said, “it's a little bit more complicated than that.” Then I had right heart catheterization and even though my numbers were very low at 27, 31, I was class 4. They said for our daughter that was getting married very soon, to move her wedding date up if they wanted me to be there. They basically gave me 6 to 9 months to survive and December 12, 2018, it'll be 16 years.

It's been very frustrating, because people aren't aware of the disease. I wear a zebra every day to help raise awareness. I developed a little card that explains the (medical) zebra analogy on one side, and a little bit about PH or pulmonary hypertension on the other side. I give those out on a regular basis. I've probably given out five or six thousand of them over the years. That's the purpose of wearing zebra every day, 365 days a year. When people look at me funny, I'll take a card out of my pocket, pants always have to have pockets, whip out a card, give it to them and say this is why I wear zebra. Just take a look at it, this will explain the zebra analogy to you a little bit and pulmonary hypertension on the other side. Then I'm on my way again, so they don't feel like they're compromised. And I'll tell cashiers if they say something about the oxygen. I'll give them a card and just say well, read this when you get a chance.

My mother-in-law died from pulmonary hypertension, as did my sister-in-law and probably a grandfather-in-law. So, it's familial on my husband's side. And then it's idiopathic on my side. People need to be aware of this, because my sister-in-law went to bed one night and didn't wake up the next morning and she was 35 years old. At the autopsy is where they saw that there was a problem with pulmonary artery. It had constricted. And they said that was the reason for her passing.

If you don't understand something ask. If you don't understand that, get on the Facebook groups. There's always somebody on Facebook that can answer your question. I have a list of places that will explain pulmonary hypertension in lay terms, as well as scientific terms or medical terms, that I send those links to different people, so they can understand what's going on with their body.

One thing that I wish I had known that a doctor didn't tell me was that this is not a death sentence. There is hope. Try to stay upbeat. Wear zebra. My mission in all of this is that when a woman feels a lump in her breast, she goes to her doctor and says could this be breast cancer. I want when a person has shortness of breath, fatigue, bluish tint to their lips, nail beds, swollen ankles, the extreme fatigue, which was my worst thing, and also hoarseness, because of the enlarged pulmonary artery. When they have those symptoms I want them to go to the doctor and say could this be pulmonary hypertension?

]]>In this episode, pulmonary hypertension patient Barbara Thompson discusses why she wears zebra clothing 365 days a year and her long road through 14 doctors before a young grad student gave her a proper PH diagnosis.

]]>04:39cleanblood,research,heart,awareness,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseaseIn this episode, pulmonary hypertension patient Barbara Thompson discusses why she wears zebra clothing 365 days a year and her long road through 14 doctors before a young grad student gave her a proper PH diagnosis.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials @antidote_me330fullphaware global associationDiane Dauwalder - phaware® interview 228Tue, 12 Mar 2019 12:00:00 +0000

Pulmonary hypertension patient Diane Dauwalder discusses how she became symptomatic in 2002, preparing for the end of her life, and how thanks to PH research and new therapies how she adjusted to a new normal.

I was confirmed diagnosed in February of 2005, but I started to become symptomatic around 2002 when I moved from Arizona to Colorado. Everybody had thought it was just the altitude and that I needed to get used to it, but all of a sudden I couldn't take stairs two at a time. I used to be a courier for Federal Express, so I was used to a fast-paced lifestyle. All of a sudden, I was getting short of breath. I knew something was wrong.

So we did move from Colorado to Fort Worth Texas and I was still having problems. I'm no longer at mile high and was still having difficulty breathing. Nobody knew what was wrong with me until I ended up in the ER. Severe sleep apnea was the first diagnosis. Then finally I was able to get to UT Southwestern, who did the right heart catheterization and confirmed my pulmonary hypertension.

It was good to know what I was dealing with, because I knew that something was wrong with me but I had no idea what. At that time, and we're talking over 10 years ago, if you went online you had three to five years to live. It was very scary. There was only two medications out. I was getting prepared for the end of my life.

At that time, I had a long talk with myself. I wasn't working obviously. At first, I was in denial, which I think we all go through. I thought they were going to give me a pill and make me better. Well, after the first six months of being on a pill and knowing I wasn't getting better, I was like, okay, time to come to terms with the Lord and make our peace here, because I've had a good life. I was ready.

Then fortunately, through research, through fundraising, more drugs came out and it kept me going. I was getting better. So I wasn't going to be in a scooter and on oxygen 24/7 anymore. I started to get better. Not much better, but I got adjusted to my new norm.

I heard about support group meetings. I didn't know anything about pulmonary hypertension. I did go to a support group meeting, and I went to a couple of them and then I walked out because I got too depressed. People who I saw walk in, the next meeting they were in wheelchairs. I just couldn't deal with it. I guess I was still in a little bit of denial.

Again, there was a third drug added to my regimen, and I was very faithful because I was on inhaled prostacyclin, which I had to do nine treatments a day every two hours, and I did it because I did not want IV therapy. From there, it was just all uphill for me.

Then I ended up becoming the support group leader. It was good. Every time that phone rings, I'm thankful that I'm doing what I'm doing, because that other person on the phone is so alone and so scared, and it's not that bad. You can have a normal life. Life can be good. You can enjoy it.

When they came out with the (Sometimes It’s PH) zebra campaign to raise awareness, which I thought was great, because it gave us an icon to use, a zebra. We are medical zebras. I got us on Good Morning Texas for our Zebra Fest, because I don't think everybody's heard of Zebra Fest that we have in North Texas. I think the zebra was a good way to raise awareness, because it made people look and listen.

My primary care physician for instance, every time I went to him he would write COPD down. I kept telling him I don't have COPD. It's PH. Even though the symptoms are the same. So finally, I started bringing him in different materials to hang on his wall, saying sometimes it's the other blood pressure. It's been good, because I think we're finally getting patients diagnosed sooner than later, which is really good because I think more people are aware of the illness. I think there's a lot more people who have pulmonary hypertension that were diagnosed incorrectly or were never really diagnosed, because nobody knew what it was.

If I met a newly diagnosed patient, I would give them a hug first of all, tell them that they're not alone, that it's a journey, but it's a good journey, and just to get used to your new normal and life goes on. It can be good.

Pulmonary hypertension patient Diane Dauwalder discusses how she became symptomatic in 2002, preparing for the end of her life, and how thanks to PH research and new therapies how she adjusted to a new normal.

I was confirmed diagnosed in February of 2005, but I started to become symptomatic around 2002 when I moved from Arizona to Colorado. Everybody had thought it was just the altitude and that I needed to get used to it, but all of a sudden I couldn't take stairs two at a time. I used to be a courier for Federal Express, so I was used to a fast-paced lifestyle. All of a sudden, I was getting short of breath. I knew something was wrong.

So we did move from Colorado to Fort Worth Texas and I was still having problems. I'm no longer at mile high and was still having difficulty breathing. Nobody knew what was wrong with me until I ended up in the ER. Severe sleep apnea was the first diagnosis. Then finally I was able to get to UT Southwestern, who did the right heart catheterization and confirmed my pulmonary hypertension.

It was good to know what I was dealing with, because I knew that something was wrong with me but I had no idea what. At that time, and we're talking over 10 years ago, if you went online you had three to five years to live. It was very scary. There was only two medications out. I was getting prepared for the end of my life.

At that time, I had a long talk with myself. I wasn't working obviously. At first, I was in denial, which I think we all go through. I thought they were going to give me a pill and make me better. Well, after the first six months of being on a pill and knowing I wasn't getting better, I was like, okay, time to come to terms with the Lord and make our peace here, because I've had a good life. I was ready.

Then fortunately, through research, through fundraising, more drugs came out and it kept me going. I was getting better. So I wasn't going to be in a scooter and on oxygen 24/7 anymore. I started to get better. Not much better, but I got adjusted to my new norm.

I heard about support group meetings. I didn't know anything about pulmonary hypertension. I did go to a support group meeting, and I went to a couple of them and then I walked out because I got too depressed. People who I saw walk in, the next meeting they were in wheelchairs. I just couldn't deal with it. I guess I was still in a little bit of denial.

Again, there was a third drug added to my regimen, and I was very faithful because I was on inhaled prostacyclin, which I had to do nine treatments a day every two hours, and I did it because I did not want IV therapy. From there, it was just all uphill for me.

Then I ended up becoming the support group leader. It was good. Every time that phone rings, I'm thankful that I'm doing what I'm doing, because that other person on the phone is so alone and so scared, and it's not that bad. You can have a normal life. Life can be good. You can enjoy it.

When they came out with the (Sometimes It’s PH) zebra campaign to raise awareness, which I thought was great, because it gave us an icon to use, a zebra. We are medical zebras. I got us on Good Morning Texas for our Zebra Fest, because I don't think everybody's heard of Zebra Fest that we have in North Texas. I think the zebra was a good way to raise awareness, because it made people look and listen.

My primary care physician for instance, every time I went to him he would write COPD down. I kept telling him I don't have COPD. It's PH. Even though the symptoms are the same. So finally, I started bringing him in different materials to hang on his wall, saying sometimes it's the other blood pressure. It's been good, because I think we're finally getting patients diagnosed sooner than later, which is really good because I think more people are aware of the illness. I think there's a lot more people who have pulmonary hypertension that were diagnosed incorrectly or were never really diagnosed, because nobody knew what it was.

If I met a newly diagnosed patient, I would give them a hug first of all, tell them that they're not alone, that it's a journey, but it's a good journey, and just to get used to your new normal and life goes on. It can be good.

]]>fullEpisode 228 - Diane DauwalderDiane Dauwalder - Pulmonary HypertensionMon, 11 Mar 2019 12:00:00 +0000In this episode, pulmonary hypertension patient Diane Dauwalder discusses how she became symptomatic in 2002, preparing for the end of her life, and how thanks to PH research and new therapies how she adjusted to a new normal.

]]>In this episode, pulmonary hypertension patient Diane Dauwalder discusses how she became symptomatic in 2002, preparing for the end of her life, and how thanks to PH research and new therapies how she adjusted to a new normal.

]]>04:05cleanblood,research,heart,awareness,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseaseIn this episode, pulmonary hypertension patient Diane Dauwalder discusses how she became symptomatic in 2002, preparing for the end of her life, and how thanks to PH research and new therapies how she adjusted to a new normal.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials @antidote_me329fullphaware global associationAlleena Shiwdas - phaware® interview 227Fri, 08 Mar 2019 13:00:00 +0000

In this episode, pulmonary hypertension patient, Alleena Shiwdas discusses the importance of a good peer support network and how psychological care as critical part of a PH treatment plan.

My name is Alleena Shiwdas, and I'm a pulmonary hypertension patient.

It was the day after I had my second son. I was in the hospital. I had a C-section and they told me I had to get up and walk. I said okay. Besides the fact that I was in a lot of pain, I was unable to breath. I couldn't walk five feet to the bathroom. I was completely out of breath. I said to them, I was like, "Something is wrong." They're like, "Um, well, everything looks fine," so I said okay. I thought I was having trouble with the medications they were giving me. I thought I had an allergic reaction, so I didn't make a big deal out of it.

After that happened, I went home and I just kept getting worst. My shortness of breath was escalating every single day. I would go to the ER. I went to multiple doctors and I said, "Something's wrong with me. I can't breathe." They're like, "Well, your stats look good. Everything's coming out good. Everything's fine. You look good. Go home." I did that for almost two years before I was finally diagnosed with primary pulmonary hypertension.

It was a week before my 24th birthday and they said, "You have less than a year to live. Your heart and lungs are in such bad shape. You're going to die of heart failure." It was definitely not a relief. I mean maybe it was, I don't know, but I was definitely in shock. I asked my sister, "Can you look up primary pulmonary hypertension on the computer for me?" I was still in the hospital. When she read the outcome, it was like everybody was dying within two years. I said to her, "That can't be right." I refused to believe it at the time. I went home and they told me I had to do a transplant. That's when they transferred me over to a different doctor and he decided, "Don't do a transplant yet. We're going to try you on therapy."

That was 11 years ago. I started therapy. I started oral therapy and within six months, I was not getting any better. The doctor put me in the best thing they had, which was IV therapy. IV therapy literally gave me my life back. I was able to breathe. I was able to live a pretty much normal life, except for swimming, and I can't really run yet. Other than that, I can do everything.

I would say learn everything from the medical terminologies to the simple things. Read everything you can. Educate yourself. Educate your family. Educate your friends. It's very important that everybody around you knows what's going on and create a network of support. Without support, you're not going anywhere. You're not going to make it. It's super important for everyone to have support.

Peer support is very important because a lot of people are diagnosed. I remember when I was diagnosed, I felt socially isolated because there was nobody in town that I thought had the same thing that I had. I am a part of every online group there is. Whenever newly diagnosed patients come on and they have questions of the same exact things that I've done in the past or experienced, I can go on there and say, "Hey, this is my experience or this is something that you can try and this is how it worked." A lot of times there are very grateful. They're like, "Oh, well, I didn't know." It's an eye opener. It's definitely helpful.

Besides being a support group leader, I think everywhere I go because I'm always red, everyone's saying to me, "Why are you always red?" I'm living on a pump, which my blood vessels are dilated 24/7. I think that's probably my biggest way of advocating for PH, yeah.

When doctors see a patient who's not in good shape, the first thing that they jump to is that you're going to die, and I don't think anyone has the right to do that. I think with the right care, therapies, treatments, patients have the opportunity to lead a normal life and live many years. PH patients are now living 20-30 years and they all had I'm sure at one point death sentences.

A lot of times when patients are diagnosed, doctors, they're always pushing therapies, breathing therapies, physical therapy, all of that, but nobody ever pushes psychological therapies. I think that's something that we ought to start implementing in the PH community, psychological care, because that's a big thing that a lot of patients are dealing with that are not addressed properly. When I was first diagnosed and they gave me this short lifespan to live, I was like, "Okay. I can't do much." Then, meeting friends who were living with PH, Sean Wyman, who recently passed, he actually pushed me to go back to school. At the time, he was in college and he told me that there's no reason that I should not go back.

I said, "You know what? That's what I want to do too," so I can learn how to deal with not only my situation, but to help others overcome what they were dealing with as well, especially in the PH community.

In this episode, pulmonary hypertension patient, Alleena Shiwdas discusses the importance of a good peer support network and how psychological care as critical part of a PH treatment plan.

My name is Alleena Shiwdas, and I'm a pulmonary hypertension patient.

It was the day after I had my second son. I was in the hospital. I had a C-section and they told me I had to get up and walk. I said okay. Besides the fact that I was in a lot of pain, I was unable to breath. I couldn't walk five feet to the bathroom. I was completely out of breath. I said to them, I was like, "Something is wrong." They're like, "Um, well, everything looks fine," so I said okay. I thought I was having trouble with the medications they were giving me. I thought I had an allergic reaction, so I didn't make a big deal out of it.

After that happened, I went home and I just kept getting worst. My shortness of breath was escalating every single day. I would go to the ER. I went to multiple doctors and I said, "Something's wrong with me. I can't breathe." They're like, "Well, your stats look good. Everything's coming out good. Everything's fine. You look good. Go home." I did that for almost two years before I was finally diagnosed with primary pulmonary hypertension.

It was a week before my 24th birthday and they said, "You have less than a year to live. Your heart and lungs are in such bad shape. You're going to die of heart failure." It was definitely not a relief. I mean maybe it was, I don't know, but I was definitely in shock. I asked my sister, "Can you look up primary pulmonary hypertension on the computer for me?" I was still in the hospital. When she read the outcome, it was like everybody was dying within two years. I said to her, "That can't be right." I refused to believe it at the time. I went home and they told me I had to do a transplant. That's when they transferred me over to a different doctor and he decided, "Don't do a transplant yet. We're going to try you on therapy."

That was 11 years ago. I started therapy. I started oral therapy and within six months, I was not getting any better. The doctor put me in the best thing they had, which was IV therapy. IV therapy literally gave me my life back. I was able to breathe. I was able to live a pretty much normal life, except for swimming, and I can't really run yet. Other than that, I can do everything.

I would say learn everything from the medical terminologies to the simple things. Read everything you can. Educate yourself. Educate your family. Educate your friends. It's very important that everybody around you knows what's going on and create a network of support. Without support, you're not going anywhere. You're not going to make it. It's super important for everyone to have support.

Peer support is very important because a lot of people are diagnosed. I remember when I was diagnosed, I felt socially isolated because there was nobody in town that I thought had the same thing that I had. I am a part of every online group there is. Whenever newly diagnosed patients come on and they have questions of the same exact things that I've done in the past or experienced, I can go on there and say, "Hey, this is my experience or this is something that you can try and this is how it worked." A lot of times there are very grateful. They're like, "Oh, well, I didn't know." It's an eye opener. It's definitely helpful.

Besides being a support group leader, I think everywhere I go because I'm always red, everyone's saying to me, "Why are you always red?" I'm living on a pump, which my blood vessels are dilated 24/7. I think that's probably my biggest way of advocating for PH, yeah. When doctors see a patient who's not in good shape, the first thing that they jump to is that you're going to die, and I don't think anyone has the right to do that. I think with the right care, therapies, treatments, patients have the opportunity to lead a normal life and live many years. PH patients are now living 20-30 years and they all had I'm sure at one point death sentences.

A lot of times when patients are diagnosed, doctors, they're always pushing therapies, breathing therapies, physical therapy, all of that, but nobody ever pushes psychological therapies. I think that's something that we ought to start implementing in the PH community, psychological care, because that's a big thing that a lot of patients are dealing with that are not addressed properly. When I was first diagnosed and they gave me this short lifespan to live, I was like, "Okay. I can't do much." Then, meeting friends who were living with PH, Sean Wyman, who recently passed, he actually pushed me to go back to school. At the time, he was in college and he told me that there's no reason that I should not go back.

I said, "You know what? That's what I want to do too," so I can learn how to deal with not only my situation, but to help others overcome what they were dealing with as well, especially in the PH community.

]]>05:00cleanblood,research,heart,awareness,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseaseIn this episode, pulmonary hypertension patient, Alleena Shiwdas discusses the importance of a good peer support network and how psychological care as critical part of a PH treatment plan.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials @antidote_me 328fullphaware global associationAlexis Trotter - phaware® interview 226Tue, 05 Mar 2019 13:00:00 +0000Pulmonary hypertension patient Alexis Trotter discusses living on oxygen 24/7, her passion for hula dancing, what inspired her to become a cardiac sonographer and her love of Camp del Corazon.

Hi. My name is Alexis Trotter, and I am a pulmonary hypertension patient.

I was born with a congenital heart defect and I had my repair when I was four years old. However, after my surgery, I remained symptomatic. The doctors told my parents that I was just doing it for attention. Finally, my mom demanded that they figure out what was wrong with me, and it was there where they saw I had peripheral pulmonary artery stenosis, which caused pulmonary hypertension.

I wasn't really on anything then because there was nothing for pediatric [patients] back then. I had lots of angioplasties to open up the narrowings, but that was my treatment. The really big crossing point for me because I had taken oral medications, they even added Tyvaso, but the thing that was really a tipping point for me was the oxygen for like as much as possible use. That was the point where pulmonary hypertension came to the forefront of my life.

When I was seven, I never realized that at age 27, I would have to traverse, starting a career, dating, like all that stuff with an oxygen tank. But to help me get through it, I just take each day at a time. I named my tank. Her name is Stella the Tank. Sometimes we get along. Sometimes we don't. It's just like baby steps. It's like each day, but I also never let pulmonary hypertension keep me from doing what I want to do.

I knew I couldn't be a nurse because it was too physical of a job for me, so I became a cardiac sonographer so I can still interact with patients, but it's not as physical as a nurse. I always wanted to do it because I spent so much time in the hospital as a kid and the nurses were just so sweet and nice to me. I'm like, "You know what? I have to pay that forward."

I found out about Camp del Corazon through my cardiologist's office and I went there. It changed my life. I thought I was the only girl with a heart surgery scar and there was other kids who had to take medicine. There were other kids who got tired really fast. I got complimented on my scar. Now that I've aged out obviously, it's been a thing of mine to give back what they have given to me because I truly believe I would not be who I was if I would not have gotten to that camp.

The name of the camp is Camp del Corazon. It is on beautiful Catalina Island. It is for children with congenital heart defects. It's staffed by nurses from UCLA. They volunteered their time. Dr. Kevin Shannon from UCLA is also there. It's like a normal camp for kids who are usually told they can't do like normal stuff. We go hiking. We go kayaking. There's a ropes course. There's dances, skit nights. It's like for those four days, us getting tired, that's normal. Like normal people, that's not normal. We're normal. It's like one little four day stretch in the summer where we feel normal.

I just try not to hold back anything. It's never been like "I can't do this." It's like "let's figure out a way for me to get it done." That's why I got a new smaller oxygen tank because the other one was on my back. It hurt my back during clinicals, so I got the small one so I could take it with me into the hospital.

I also am a dancer for fun. I was always really self-conscious about wearing it in dance class for some reason, but my hula sister, she made this little backpack for my tank and I wear it for our shows. I've done a couple competitions. It's always like my mindset is how can I get this done, not like I can't do it, but how can we work around it.

My advice to you is that it's alright to feel at times that it's unfair because it is unfair, and you didn't do anything to get what you're going through, but don't sit in that. You have to pick yourself up and move forward. Find yourself a good support group. I'm not saying you have to find other people with pulmonary hypertension or whatever, because I'm the only person in my family who has it, but I have good friends. I have a good family. Don't dwell on the fact that you have this issue. Appreciate the good things that you do have.

Like I said, I have a good family. I went to good schools. I travel. I love to shop. I get to do all these other things, and I don't let that be my main focus. Just rework your mind. Think not that I can't do it, but how can you do it. You know? If you want to do a 5K, you probably can't run it, but you could certainly walk it. You can go to pulmonary rehab and they'll get you conditioned to do it.

]]>Pulmonary hypertension patient Alexis Trotter discusses living on oxygen 24/7, her passion for hula dancing, what inspired her to become a cardiac sonographer and her love of Camp del Corazon.

Hi. My name is Alexis Trotter, and I am a pulmonary hypertension patient.

I was born with a congenital heart defect and I had my repair when I was four years old. However, after my surgery, I remained symptomatic. The doctors told my parents that I was just doing it for attention. Finally, my mom demanded that they figure out what was wrong with me, and it was there where they saw I had peripheral pulmonary artery stenosis, which caused pulmonary hypertension.

I wasn't really on anything then because there was nothing for pediatric [patients] back then. I had lots of angioplasties to open up the narrowings, but that was my treatment. The really big crossing point for me because I had taken oral medications, they even added Tyvaso, but the thing that was really a tipping point for me was the oxygen for like as much as possible use. That was the point where pulmonary hypertension came to the forefront of my life.

When I was seven, I never realized that at age 27, I would have to traverse, starting a career, dating, like all that stuff with an oxygen tank. But to help me get through it, I just take each day at a time. I named my tank. Her name is Stella the Tank. Sometimes we get along. Sometimes we don't. It's just like baby steps. It's like each day, but I also never let pulmonary hypertension keep me from doing what I want to do.

I knew I couldn't be a nurse because it was too physical of a job for me, so I became a cardiac sonographer so I can still interact with patients, but it's not as physical as a nurse. I always wanted to do it because I spent so much time in the hospital as a kid and the nurses were just so sweet and nice to me. I'm like, "You know what? I have to pay that forward."

I found out about Camp del Corazon through my cardiologist's office and I went there. It changed my life. I thought I was the only girl with a heart surgery scar and there was other kids who had to take medicine. There were other kids who got tired really fast. I got complimented on my scar. Now that I've aged out obviously, it's been a thing of mine to give back what they have given to me because I truly believe I would not be who I was if I would not have gotten to that camp.

The name of the camp is Camp del Corazon. It is on beautiful Catalina Island. It is for children with congenital heart defects. It's staffed by nurses from UCLA. They volunteered their time. Dr. Kevin Shannon from UCLA is also there. It's like a normal camp for kids who are usually told they can't do like normal stuff. We go hiking. We go kayaking. There's a ropes course. There's dances, skit nights. It's like for those four days, us getting tired, that's normal. Like normal people, that's not normal. We're normal. It's like one little four day stretch in the summer where we feel normal.

I just try not to hold back anything. It's never been like "I can't do this." It's like "let's figure out a way for me to get it done." That's why I got a new smaller oxygen tank because the other one was on my back. It hurt my back during clinicals, so I got the small one so I could take it with me into the hospital.

I also am a dancer for fun. I was always really self-conscious about wearing it in dance class for some reason, but my hula sister, she made this little backpack for my tank and I wear it for our shows. I've done a couple competitions. It's always like my mindset is how can I get this done, not like I can't do it, but how can we work around it. My advice to you is that it's alright to feel at times that it's unfair because it is unfair, and you didn't do anything to get what you're going through, but don't sit in that. You have to pick yourself up and move forward. Find yourself a good support group. I'm not saying you have to find other people with pulmonary hypertension or whatever, because I'm the only person in my family who has it, but I have good friends. I have a good family. Don't dwell on the fact that you have this issue. Appreciate the good things that you do have.

Like I said, I have a good family. I went to good schools. I travel. I love to shop. I get to do all these other things, and I don't let that be my main focus. Just rework your mind. Think not that I can't do it, but how can you do it. You know? If you want to do a 5K, you probably can't run it, but you could certainly walk it. You can go to pulmonary rehab and they'll get you conditioned to do it.

]]>In this episode, pulmonary hypertension patient Alexis Trotter discusses living on oxygen 24/7, her passion for hula dancing, what inspired her to become a cardiac sonographer and her love of Camp del Corazon.

In this episode, 40 year long-term pulmonary hypertension survivor Gwendolyn R. Brown discusses the importance of clinical trials, building a great PH care team, and how THE LION KING is a perfect metaphor for PH.

Hi, my name is Gwendolyn R. Brown, and I am a 40 year survivor with pulmonary hypertension.

When I was diagnosed at that time, there was nothing out there. For three years prior, I was getting sick, I thought it was a cold. And I kept going to the doctor, and then they diagnosed me with asthma. The asthma medication wasn’t working, and it started getting worse and worse. I started passing out. And my last time I passed out, I remember waking up in the ICU and there was this one doctor there. And he was like, "I think you have this disease. It's a rare disease, but I need to find out." Back then, they didn't do right heart catheterization, they did lung biopsies.

So, I had to have a lung biopsy done, and when he came back, he said, "You have pulmonary hypertension." He said, "It's a very rare lung disease." He said, "We don't even know nothing about it right now." He said, "All I can tell you is go home and get your life in order." He said, "I don't expect you to live two years with this."

And I did, I went home and got my life in order. I did research, and I just took care of myself. I ate healthy, I lost the weight, and walked every day. I did everything my doctor told me to do. And that lasted until 1998, when Flolan came out, and I was picked as one of the trials to go try it out at Duke University. And that's where I met the great Dr. Victor Tapson. He introduced me to Flolan, and from then on it was amazing.

I think [clinical trials] are important for patients, because even though the trial might not help me, it can help someone else. With the disease being as rare as it was, I put my name down for every trial. I am so amazed when I see all the medicine, all the treatments that are available now for people. It's just astounded me. I end up crying because it's just beautiful to see it all. But like I said, when I was coming up, there was nothing. And when they would start new trials, I was like, "I'm here. Can you help me?" And with the Flolan, at that time I was on oxygen all day. I was in a wheelchair. I was on the heart and lung transplant, because back in those days, they were only going to both lung and heart transplant.

And I was like, "No, I don't want that." But then they came out with Flolan and I went and had the trial. And immediately [when I went on Flolan] I was off oxygen. So, I think trials are great for everyone to try out, and research, I don't think they should stop research. More money should be invested into it, to see if we can't cure it, at least put it in recession. Get it to stop growing.

Being on 40 years of surviving and watching people, I feel amazed, I feel gifted to see it. I really do. Just to be here, to see people that I can help, that I can tell them, "You can do this, you can get through this. It don't define who you are." We all have to live through our tests and trials. I tell people, "I have PH, PH don't have me." I'm going to live my life. And I like when they say, "You lived 40 years, who do you do it?" I say, "One step at a time, with my faith. And you have to have a positive attitude, because if you sit depressed, you're going to be depressed. If you be happy, you can make it through it."

And to see other people doing what they're doing, living what they're doing. I tell them do what your body says you can do. I rode a bike, I was crying on that bike ride and people thought I was going crazy. But every step. Do what you can do, what your body says you can do. But always take care of it.

I think when you're first diagnosed, make sure you have a great team. And that's including your doctor, your specialist, your nurse, your caregiver. I love the movie The Lion King, because it tells you about the circle of life. And all these events go with this circle. And you end up right back where you started from at the end, and that's how I look at it with PH.

It's a circle of life that we have to start with our doctors. But then eventually it comes down to us, that we take over and start taking care of us. But we keep that circle going, and I think that with a new person, listen to your doctor. Listen to your body. Hang around positive people, people that encourage you, not people to put you down. And I think that will help you be able to handle this disease and live with it.

In this episode, 40 year long-term pulmonary hypertension survivor Gwendolyn R. Brown discusses the importance of clinical trials, building a great PH care team, and how THE LION KING is a perfect metaphor for PH.

Hi, my name is Gwendolyn R. Brown, and I am a 40 year survivor with pulmonary hypertension.

When I was diagnosed at that time, there was nothing out there. For three years prior, I was getting sick, I thought it was a cold. And I kept going to the doctor, and then they diagnosed me with asthma. The asthma medication wasn’t working, and it started getting worse and worse. I started passing out. And my last time I passed out, I remember waking up in the ICU and there was this one doctor there. And he was like, "I think you have this disease. It's a rare disease, but I need to find out." Back then, they didn't do right heart catheterization, they did lung biopsies.

So, I had to have a lung biopsy done, and when he came back, he said, "You have pulmonary hypertension." He said, "It's a very rare lung disease." He said, "We don't even know nothing about it right now." He said, "All I can tell you is go home and get your life in order." He said, "I don't expect you to live two years with this."

And I did, I went home and got my life in order. I did research, and I just took care of myself. I ate healthy, I lost the weight, and walked every day. I did everything my doctor told me to do. And that lasted until 1998, when Flolan came out, and I was picked as one of the trials to go try it out at Duke University. And that's where I met the great Dr. Victor Tapson. He introduced me to Flolan, and from then on it was amazing.

I think [clinical trials] are important for patients, because even though the trial might not help me, it can help someone else. With the disease being as rare as it was, I put my name down for every trial. I am so amazed when I see all the medicine, all the treatments that are available now for people. It's just astounded me. I end up crying because it's just beautiful to see it all. But like I said, when I was coming up, there was nothing. And when they would start new trials, I was like, "I'm here. Can you help me?" And with the Flolan, at that time I was on oxygen all day. I was in a wheelchair. I was on the heart and lung transplant, because back in those days, they were only going to both lung and heart transplant.

And I was like, "No, I don't want that." But then they came out with Flolan and I went and had the trial. And immediately [when I went on Flolan] I was off oxygen. So, I think trials are great for everyone to try out, and research, I don't think they should stop research. More money should be invested into it, to see if we can't cure it, at least put it in recession. Get it to stop growing.

Being on 40 years of surviving and watching people, I feel amazed, I feel gifted to see it. I really do. Just to be here, to see people that I can help, that I can tell them, "You can do this, you can get through this. It don't define who you are." We all have to live through our tests and trials. I tell people, "I have PH, PH don't have me." I'm going to live my life. And I like when they say, "You lived 40 years, who do you do it?" I say, "One step at a time, with my faith. And you have to have a positive attitude, because if you sit depressed, you're going to be depressed. If you be happy, you can make it through it."

And to see other people doing what they're doing, living what they're doing. I tell them do what your body says you can do. I rode a bike, I was crying on that bike ride and people thought I was going crazy. But every step. Do what you can do, what your body says you can do. But always take care of it.

I think when you're first diagnosed, make sure you have a great team. And that's including your doctor, your specialist, your nurse, your caregiver. I love the movie The Lion King, because it tells you about the circle of life. And all these events go with this circle. And you end up right back where you started from at the end, and that's how I look at it with PH.

It's a circle of life that we have to start with our doctors. But then eventually it comes down to us, that we take over and start taking care of us. But we keep that circle going, and I think that with a new person, listen to your doctor. Listen to your body. Hang around positive people, people that encourage you, not people to put you down. And I think that will help you be able to handle this disease and live with it.

]]>In this episode, 40 year long-term pulmonary hypertension survivor Gwendolyn R. Brown discusses the importance of clinical trials, building a great PH care team, and how THE LION KING is a perfect metaphor for PH.

]]>04:38cleanblood,research,heart,awareness,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdisease40 year long-term pulmonary hypertension survivor Gwendolyn R. Brown discusses the importance of clinical trials, building a great PH care team, and how THE LION KING is a perfect metaphor for PH.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware #ClinicalTrials @antidote_me326fullphaware global associationJudith Moatti - phaware® interview 224Tue, 26 Feb 2019 13:00:00 +0000

In this episode, PHA Canada ambassador Judith Moatti discusses the importance of early diagnosis and awareness. Judith was first diagnosed with Pulmonary Arterial Hypertension (PAH) in 2009, at the young age of 25. After a long road to stability and coping with her new reality, she has become a strong advocate and spokesperson for those affected by PAH. Locally, she became a patient representative for Foundation HTAPQ (a Québec-based patient support organization) in 2012. And she also works as a support group leader for patients living or affected by PH in her region.

Hi, my name is Judith Moatti, and I'm a pulmonary hypertension patient.

When I first got diagnosed, I got my diagnosis by fluke. My gallbladder got swelled up, and I went to [the ER], because it was hurting a lot and I could barely walk anymore. When I got to emergency, they thought I had stones, but they couldn't find anything. They wanted to remove my gallbladder. In the process, my mom refused to let them take me for surgery because she thought it wasn't reasonable to take out my gallbladder if we didn't know why it was swelled up.

I had to go through a bunch of autoimmune disease tests, and [my doctor] realized that I didn't have any autoimmune disease. They were going to send me home. So, I'm waiting for the doctor with the release form. As I was waiting in her office, I saw a poster on the wall that described pulmonary hypertension and the symptoms. My friend who was with me told her to test me for that because I had been short of breath when I was walking.

We started with the chest x-ray, and she said my lung arteries looked a little small, but that it might not mean anything. Then we went for a heart ultrasound. That's when my life changed for the worst in a way. I started with one technician in radiology. I ended up with 15 doctors and residents in the room because I was in heart failure. That's when they announced to me that I had pulmonary hypertension.

They transferred me to the cardiologist specialist at the Montreal Jewish Hospital and as soon as I met with him, he explained to me that I was really close to death, and that I had to quit everything I was doing. I had to quit my job. I couldn't have children anymore. We started on IV therapy right away because there was nothing else that was working in bringing my pressures down. It was a difficult therapy, of course. After a while, I had to be switched therapies because the site pain never left in my case.

After a few years of switching therapies and going on research protocols, I was put on all oral therapies. I tried to go back to work. My pressure was going back up, so my doctor suggested that I completely stop working. If not, I was going to die young. When he stopped me from working, I was wondering what can I do to help other people? So I started to host support groups. Then, there's also a foundation in Quebec, HTAPQ, and we help patients in our province. They asked me to be part of the board, and so I became a board member. Through all these activities, I met PHA Canada, because as soon as we are diagnosed and we become a member of the other foundation, we become a member with PHA Canada.

I've always wanted to be involved with them, but I had to take it slow because I didn't know everything, and I didn't know how I was going to help people. Eventually, I started going to their conferences, which are really good conferences. I met a lot of different people. I realized I wasn't alone and that I could help others like me.

Not long ago, I saw that there was a post on Facebook where there was an opening for an ambassador role, and right away I applied, went through an interview, and was chosen to actually be a part of that. I'm really glad I am because I can help more people through PHA Canada because it's on a national level.

I want to be an ambassador because I want to help other people know about pulmonary hypertension. I think not a lot of people know what it is and how it affects us on a daily basis. I think it's important to help others as well. When I got my diagnosis, I felt very alone. When I started following PHA Canada, I realized that there was a lot of other people like me in Canada that were having pulmonary hypertension, that had the same issues as me. I definitely wanted to help them because I went through a hard, hard time, and I know how I got out of it. So I know how to help others and share my story with other so that they can understand that they're not alone either.

We're trying to make people aware and doctors especially, like emergency doctors, general practitioners, we're trying to teach them about pulmonary hypertension because oftentimes, people go to their family doctor and they're misdiagnosed. In my case, it was the same thing. I went to my family doctor who told me it was normal that I would faint once in a while. Going through this experience, I knew that I wanted to help others not go through the same, that it's really important to get an early diagnosis because people can potentially keep working and have somewhat of a normal life even with pulmonary hypertension.

So it's really important for me to try and reach those people because they need to understand that someone [who presents with] shortness of breath, it doesn't necessarily mean they have asthma or that they're not training enough. It's really important to teach them as well that there's something bigger that you might want to check out before you send someone home and tell them that they're normal and everything is fine.

There's so much that changed throughout the last years. I've had my diagnosis for eight years now, and in the little bit of eight years that I've been sick, I've seen a lot of changes. There's been a lot of new therapies, a lot more support groups, a lot more people are sharing their stories now, which I find is wonderful because this is how we're going to get the word across.

In this episode, PHA Canada ambassador Judith Moatti discusses the importance of early diagnosis and awareness. Judith was first diagnosed with Pulmonary Arterial Hypertension (PAH) in 2009, at the young age of 25. After a long road to stability and coping with her new reality, she has become a strong advocate and spokesperson for those affected by PAH. Locally, she became a patient representative for Foundation HTAPQ (a Québec-based patient support organization) in 2012. And she also works as a support group leader for patients living or affected by PH in her region.

Hi, my name is Judith Moatti, and I'm a pulmonary hypertension patient.

When I first got diagnosed, I got my diagnosis by fluke. My gallbladder got swelled up, and I went to [the ER], because it was hurting a lot and I could barely walk anymore. When I got to emergency, they thought I had stones, but they couldn't find anything. They wanted to remove my gallbladder. In the process, my mom refused to let them take me for surgery because she thought it wasn't reasonable to take out my gallbladder if we didn't know why it was swelled up.

I had to go through a bunch of autoimmune disease tests, and [my doctor] realized that I didn't have any autoimmune disease. They were going to send me home. So, I'm waiting for the doctor with the release form. As I was waiting in her office, I saw a poster on the wall that described pulmonary hypertension and the symptoms. My friend who was with me told her to test me for that because I had been short of breath when I was walking.

We started with the chest x-ray, and she said my lung arteries looked a little small, but that it might not mean anything. Then we went for a heart ultrasound. That's when my life changed for the worst in a way. I started with one technician in radiology. I ended up with 15 doctors and residents in the room because I was in heart failure. That's when they announced to me that I had pulmonary hypertension.

They transferred me to the cardiologist specialist at the Montreal Jewish Hospital and as soon as I met with him, he explained to me that I was really close to death, and that I had to quit everything I was doing. I had to quit my job. I couldn't have children anymore. We started on IV therapy right away because there was nothing else that was working in bringing my pressures down. It was a difficult therapy, of course. After a while, I had to be switched therapies because the site pain never left in my case.

After a few years of switching therapies and going on research protocols, I was put on all oral therapies. I tried to go back to work. My pressure was going back up, so my doctor suggested that I completely stop working. If not, I was going to die young. When he stopped me from working, I was wondering what can I do to help other people? So I started to host support groups. Then, there's also a foundation in Quebec, HTAPQ, and we help patients in our province. They asked me to be part of the board, and so I became a board member. Through all these activities, I met PHA Canada, because as soon as we are diagnosed and we become a member of the other foundation, we become a member with PHA Canada.

I've always wanted to be involved with them, but I had to take it slow because I didn't know everything, and I didn't know how I was going to help people. Eventually, I started going to their conferences, which are really good conferences. I met a lot of different people. I realized I wasn't alone and that I could help others like me.

Not long ago, I saw that there was a post on Facebook where there was an opening for an ambassador role, and right away I applied, went through an interview, and was chosen to actually be a part of that. I'm really glad I am because I can help more people through PHA Canada because it's on a national level.

I want to be an ambassador because I want to help other people know about pulmonary hypertension. I think not a lot of people know what it is and how it affects us on a daily basis. I think it's important to help others as well. When I got my diagnosis, I felt very alone. When I started following PHA Canada, I realized that there was a lot of other people like me in Canada that were having pulmonary hypertension, that had the same issues as me. I definitely wanted to help them because I went through a hard, hard time, and I know how I got out of it. So I know how to help others and share my story with other so that they can understand that they're not alone either.

We're trying to make people aware and doctors especially, like emergency doctors, general practitioners, we're trying to teach them about pulmonary hypertension because oftentimes, people go to their family doctor and they're misdiagnosed. In my case, it was the same thing. I went to my family doctor who told me it was normal that I would faint once in a while. Going through this experience, I knew that I wanted to help others not go through the same, that it's really important to get an early diagnosis because people can potentially keep working and have somewhat of a normal life even with pulmonary hypertension.

So it's really important for me to try and reach those people because they need to understand that someone [who presents with] shortness of breath, it doesn't necessarily mean they have asthma or that they're not training enough. It's really important to teach them as well that there's something bigger that you might want to check out before you send someone home and tell them that they're normal and everything is fine.

There's so much that changed throughout the last years. I've had my diagnosis for eight years now, and in the little bit of eight years that I've been sick, I've seen a lot of changes. There's been a lot of new therapies, a lot more support groups, a lot more people are sharing their stories now, which I find is wonderful because this is how we're going to get the word across.

]]>blood,canada,research,heart,awareness,asthma,iv,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseasefullEpisode 224 - Judith MoattiJudith Moatti - Pulmonary HypertensionMon, 25 Feb 2019 13:00:00 +0000In this episode, PHA Canada ambassador Judith Moatti discusses the importance of early diagnosis and awareness. Judith was first diagnosed with Pulmonary Arterial Hypertension (PAH) in 2009, at the young age of 25. After a long road to stability and coping with her new reality, she has become a strong advocate and spokesperson for those affected by PAH. Locally, she became a patient representative for Foundation HTAPQ (a Québec-based patient support organization) in 2012. And she also works as a support group leader for patients living or affected by PH in her region.

]]>In this episode, PHA Canada ambassador Judith Moatti discusses the importance of early diagnosis and awareness. Judith was first diagnosed with Pulmonary Arterial Hypertension (PAH) in 2009, at the young age of 25. After a long road to stability and coping with her new reality, she has become a strong advocate and spokesperson for those affected by PAH. Locally, she became a patient representative for Foundation HTAPQ (a Québec-based patient support organization) in 2012. And she also works as a support group leader for patients living or affected by PH in her region.

]]>05:24cleanblood,canada,research,heart,awareness,asthma,iv,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseasePHA Canada ambassador Judith Moatti discusses the importance of early diagnosis and awareness. Judith was first diagnosed with Pulmonary Arterial Hypertension (PAH) in 2009, at the young age of 25. After a long road to stability and coping with her new reality, she has become a strong advocate and spokesperson for those affected by PAH.
Learn more about pulmonary hypertension at www.phaware.global. Follow us @phaware Engage for a cure: www.phaware.global/donate #phaware
325fullphaware global associationKathleen Richardson - phaware® interview 223Fri, 22 Feb 2019 13:00:00 +0000

It was after my son was born, my youngest son, he was about two years old, and I was feeling some shortness of breath. It seemed odd to me, but it wasn't significant. I just figured it was after having three children and feeling a little bit short of breath. But then I went to SeaWorld with some friends, and we climbed all the way from the bottom to the top of this stadium, and I was so short of breath I didn't think I was going to recover. But I did. It was short, but I looked around and no one else was breathing as heavy as I was. So I realized there really was something wrong, and I needed to get to the doctor.

So I went to the doctor, and it took about six months going to test after test after test. They were coming back normal. The doctor asked me if I'd ever been diagnosed with panic attacks, because I think he was thinking maybe it was in my head. I don't know. That's the message I got anyway. I said, "No, it's not panic attacks," but I did start to question myself at that point and wondered whether I should keep looking because maybe there wasn't anything wrong. It was just in my head. But I did keep pursuing, and they finally did a stress test. When they did that stress test, my blood oxygen saturation dropped significantly. It went down to 78. So then they said, "Yeah, there's something wrong with you." And I said, "Yeah, I kind of figured that."

So then I had a few more tests, and then finally had a heart catheterization. After the heart catheterization, the cardiologist said, "There's nothing wrong with your heart. It's got to be something with your lungs. We did notice a little elevation in pressure over here, but not too much." Then a couple days later, he called me back and asked me to come to his office. It was there that he told me he consulted with another cardiologist and said he thought I had pulmonary hypertension. And he didn't really tell me what it was, he just said, "You need to go to a cutting edge hospital. I don't know anything about this disease, and it's important for you to get to somewhere where they know about it."

Back then, there were three treatments for pulmonary hypertension. I don't feel like there was a huge hopeful outlook. But over these last 17 years, they've developed 14 treatments for pulmonary hypertension, and it feels so much more hopeful. I feel like awareness has increased. I feel like there's still a lot to be done, but there's a lot more awareness, and I think there's more organizations that reach out to pulmonary hypertension patients. I feel like it's a lot more hopeful now.

My life really did not change very much. I thought that it was going to, because when I learned what pulmonary hypertension was, at that time they were saying that the survival rate after diagnosis was like two to eight years average. There was a lot of emotional, psychological things to kind of work through. I had little kids. My youngest was two, my oldest was seven. So just kind of trying to deal with my own mortality really. But I responded very well to the medication that I was put on.

I began to exercise. The doctor told me that I could get my heart rate up to 115, and that would still be kind of safe for me. Because above that, I would be doing more harm than good. So I started riding my bike, and I've gotten better in my pulmonary pressure. Dropped down to normal after a few years, and I've stayed real active and gotten involved in Team PHenomenal Hope. Because they started out as cyclists, and that was really exciting for me. So I've been involved with them for now the last four years. I'm on the team as an athlete, and I am a cyclist. But I've now kind of branched off into triathlons. So I've done a couple of sprint tris and I did an Olympic tri, and I'm hoping to do a half Ironman. I have a partner, a #LetMeBeYourLung partner. Haley York is my partner.

Pulmonary hypertension awareness is important because we need to get diagnosed early. There are still so many people who go for a long time without getting diagnosed or they get misdiagnosed. So it's really important for doctors to become more aware, medical professionals to become more aware. Because it's still a rare disease, and people need to know about it so they get diagnosed early, get proper treatment early. I would say, really work hard to become knowledgeable about pulmonary hypertension. Get involved in a support group. Follow your doctor's orders.

Research and trials is critical, because there's still so much that they don't know about pulmonary hypertension, so much that still needs to be learned. We want a cure. We want to get rid of this whole disease.

It was after my son was born, my youngest son, he was about two years old, and I was feeling some shortness of breath. It seemed odd to me, but it wasn't significant. I just figured it was after having three children and feeling a little bit short of breath. But then I went to SeaWorld with some friends, and we climbed all the way from the bottom to the top of this stadium, and I was so short of breath I didn't think I was going to recover. But I did. It was short, but I looked around and no one else was breathing as heavy as I was. So I realized there really was something wrong, and I needed to get to the doctor.

So I went to the doctor, and it took about six months going to test after test after test. They were coming back normal. The doctor asked me if I'd ever been diagnosed with panic attacks, because I think he was thinking maybe it was in my head. I don't know. That's the message I got anyway. I said, "No, it's not panic attacks," but I did start to question myself at that point and wondered whether I should keep looking because maybe there wasn't anything wrong. It was just in my head. But I did keep pursuing, and they finally did a stress test. When they did that stress test, my blood oxygen saturation dropped significantly. It went down to 78. So then they said, "Yeah, there's something wrong with you." And I said, "Yeah, I kind of figured that."

So then I had a few more tests, and then finally had a heart catheterization. After the heart catheterization, the cardiologist said, "There's nothing wrong with your heart. It's got to be something with your lungs. We did notice a little elevation in pressure over here, but not too much." Then a couple days later, he called me back and asked me to come to his office. It was there that he told me he consulted with another cardiologist and said he thought I had pulmonary hypertension. And he didn't really tell me what it was, he just said, "You need to go to a cutting edge hospital. I don't know anything about this disease, and it's important for you to get to somewhere where they know about it."

Back then, there were three treatments for pulmonary hypertension. I don't feel like there was a huge hopeful outlook. But over these last 17 years, they've developed 14 treatments for pulmonary hypertension, and it feels so much more hopeful. I feel like awareness has increased. I feel like there's still a lot to be done, but there's a lot more awareness, and I think there's more organizations that reach out to pulmonary hypertension patients. I feel like it's a lot more hopeful now.

My life really did not change very much. I thought that it was going to, because when I learned what pulmonary hypertension was, at that time they were saying that the survival rate after diagnosis was like two to eight years average. There was a lot of emotional, psychological things to kind of work through. I had little kids. My youngest was two, my oldest was seven. So just kind of trying to deal with my own mortality really. But I responded very well to the medication that I was put on.

I began to exercise. The doctor told me that I could get my heart rate up to 115, and that would still be kind of safe for me. Because above that, I would be doing more harm than good. So I started riding my bike, and I've gotten better in my pulmonary pressure. Dropped down to normal after a few years, and I've stayed real active and gotten involved in Team PHenomenal Hope. Because they started out as cyclists, and that was really exciting for me. So I've been involved with them for now the last four years. I'm on the team as an athlete, and I am a cyclist. But I've now kind of branched off into triathlons. So I've done a couple of sprint tris and I did an Olympic tri, and I'm hoping to do a half Ironman. I have a partner, a #LetMeBeYourLung partner. Haley York is my partner.

Pulmonary hypertension awareness is important because we need to get diagnosed early. There are still so many people who go for a long time without getting diagnosed or they get misdiagnosed. So it's really important for doctors to become more aware, medical professionals to become more aware. Because it's still a rare disease, and people need to know about it so they get diagnosed early, get proper treatment early. I would say, really work hard to become knowledgeable about pulmonary hypertension. Get involved in a support group. Follow your doctor's orders.

Research and trials is critical, because there's still so much that they don't know about pulmonary hypertension, so much that still needs to be learned. We want a cure. We want to get rid of this whole disease.

Pulmonary hypertension patient Merle Reeseman discusses her PH diagnosis and the importance of being seen by a PH specialist who understands the disease and can diagnose and prescribe the proper medication.

I'm Merle Reeseman and a pulmonary hypertension patient.

It was back probably in the late 90s. I wasn't feeling well, I was a very active person [who] went from this doctor to the next doctor. And one doctor says, "You need to see a specialist." I went to a cardiologist and he said, “whoever read this EKG, or echo, doesn't know what they're talking about. You need to go to the Cleveland Clinic.” That's where I went.

When I first went to the clinic, they said, “you have a clean body,” which meant I was not on any kind of medications. And would I mind trying to this and mind trying that. They went through a lot of trials, and I got to the point where they said, “that's it, you need to be on medicine,” because they first tell you [that] you have two to six years to live without medications. And too many patients don't hear the without medication.

So, my two to six years went to two to three months. And then they put me on the only IV medication that was available at the time. The only medication that was available at the time. And I was on that for about 10 years, and then we decided because of the distance from my home to the clinic, (which is two and a half hours), and only has what I call a 15 minute shelf life. So, I had to be put on a different IV medication. Now there are three IV medications. There’s 14 medications now available. And as I mentioned, there was only one when I was first diagnosed.

My one doctor said he's going to change Murphy's law to Merle's law, because if anything going to go wrong I would be right in the middle of it. And it's proven to be true. I just happen to be one of those people that if something's going to go askew, I'm there. I went for a right heart cath, and I had a fellow, not quite a specialist, but a doctor, and he went up through my heart for the catheterization, and he went up this much further. And he found an anonymous pulmonary vein return. Your blood goes from your heart up through your lungs, back down into the left side of your heart, and throughout your body with fresh oxygenated blood. Well, mine didn't. It came back down into the right side of my heart, which caused the right side of my heart to enlarge, which basically gave me the PH.

So, rather than having idiopathic PH, (and I always said I had to be an idiot to get it), I had a cause, a reason. So, I had open heart surgery, and they said I'd be in the hospital four to six days. Three months later, I was sent home. I could not walk, I could not do much of anything. Had rehab and everything at home for six months. Then I got out the door and went to PT and all that for another six months. So I lost a whole year there. But I'm here.

People think well a specialist is a pulmonologist, or a cardiologist. Sure, they're specialists for certain things, but they may not be a PH specialist. And that's the difference. We need to find a PH specialist who understands the disease and can diagnose and prescribe the proper medication. I mean now there's 14 medications out there with more on the way.

Years ago, I was doing some search on the internet, and the local TV station had a little thing, if you have some kind of a disease and would like to tell us about it, contact us. So I called, and I just wrote, I said I have this rare disease, they've told me I have three to five years to live. And I stopped right there. I got a phone call. I mean if you make it sound scary, they will call you. So, I had the TV reporter come. I showed them all my medicine, how I have to line it up, how I have to mix this, and do that, and everything. And I said, "Can I say this on TV?" And she said, "What?" And I said, "Well I don't want people to end up like me, with a hose in the nose and a tube in the boob." That went viral.

I want to leave you with an exert of a poem that I wrote. I used to be healthy, I used to be strong. I used to work most all the day long. And then it hit me without even a clue, I wasn't quite sure just what I could do. I saw many doctors, I had many tests, and then I was diagnosed with IPAH, and you know the rest. Now, there's three pages, and I'm not going to go through all that.

But that gives you an idea, because I was healthy, I was strong, I did work all the day long. But, you know, now I can't. You go from here to here in little or no time at all. And to some people it's overwhelming, they don't understand why, and that's when you need to ask your doctor, "Why? Why is this happening? What is this disease about?" And then learn as much as you can.

Now, a lot of people said they don't go on the [world wide web], well some people need to. But you have to get an updated version. Check the dates on anything that's on the internet. And last but not least, remember to smile, because smiles are contagious you see. And when you feel down and put on a frown, turn that frown upside down, just for me. Because smiles make us happy and will help us cope with having this dastardly pulmonary hypertension disease.

Pulmonary hypertension patient Merle Reeseman discusses her PH diagnosis and the importance of being seen by a PH specialist who understands the disease and can diagnose and prescribe the proper medication.

I'm Merle Reeseman and a pulmonary hypertension patient. It was back probably in the late 90s. I wasn't feeling well, I was a very active person [who] went from this doctor to the next doctor. And one doctor says, "You need to see a specialist." I went to a cardiologist and he said, “whoever read this EKG, or echo, doesn't know what they're talking about. You need to go to the Cleveland Clinic.” That's where I went. When I first went to the clinic, they said, “you have a clean body,” which meant I was not on any kind of medications. And would I mind trying to this and mind trying that. They went through a lot of trials, and I got to the point where they said, “that's it, you need to be on medicine,” because they first tell you [that] you have two to six years to live without medications. And too many patients don't hear the without medication. So, my two to six years went to two to three months. And then they put me on the only IV medication that was available at the time. The only medication that was available at the time. And I was on that for about 10 years, and then we decided because of the distance from my home to the clinic, (which is two and a half hours), and only has what I call a 15 minute shelf life. So, I had to be put on a different IV medication. Now there are three IV medications. There’s 14 medications now available. And as I mentioned, there was only one when I was first diagnosed. My one doctor said he's going to change Murphy's law to Merle's law, because if anything going to go wrong I would be right in the middle of it. And it's proven to be true. I just happen to be one of those people that if something's going to go askew, I'm there. I went for a right heart cath, and I had a fellow, not quite a specialist, but a doctor, and he went up through my heart for the catheterization, and he went up this much further. And he found an anonymous pulmonary vein return. Your blood goes from your heart up through your lungs, back down into the left side of your heart, and throughout your body with fresh oxygenated blood. Well, mine didn't. It came back down into the right side of my heart, which caused the right side of my heart to enlarge, which basically gave me the PH. So, rather than having idiopathic PH, (and I always said I had to be an idiot to get it), I had a cause, a reason. So, I had open heart surgery, and they said I'd be in the hospital four to six days. Three months later, I was sent home. I could not walk, I could not do much of anything. Had rehab and everything at home for six months. Then I got out the door and went to PT and all that for another six months. So I lost a whole year there. But I'm here. People think well a specialist is a pulmonologist, or a cardiologist. Sure, they're specialists for certain things, but they may not be a PH specialist. And that's the difference. We need to find a PH specialist who understands the disease and can diagnose and prescribe the proper medication. I mean now there's 14 medications out there with more on the way. Years ago, I was doing some search on the internet, and the local TV station had a little thing, if you have some kind of a disease and would like to tell us about it, contact us. So I called, and I just wrote, I said I have this rare disease, they've told me I have three to five years to live. And I stopped right there. I got a phone call. I mean if you make it sound scary, they will call you. So, I had the TV reporter come. I showed them all my medicine, how I have to line it up, how I have to mix this, and do that, and everything. And I said, "Can I say this on TV?" And she said, "What?" And I said, "Well I don't want people to end up like me, with a hose in the nose and a tube in the boob." That went viral. I want to leave you with an exert of a poem that I wrote. I used to be healthy, I used to be strong. I used to work most all the day long. And then it hit me without even a clue, I wasn't quite sure just what I could do. I saw many doctors, I had many tests, and then I was diagnosed with IPAH, and you know the rest. Now, there's three pages, and I'm not going to go through all that. But that gives you an idea, because I was healthy, I was strong, I did work all the day long. But, you know, now I can't. You go from here to here in little or no time at all. And to some people it's overwhelming, they don't understand why, and that's when you need to ask your doctor, "Why? Why is this happening? What is this disease about?" And then learn as much as you can. Now, a lot of people said they don't go on the [world wide web], well some people need to. But you have to get an updated version. Check the dates on anything that's on the internet. And last but not least, remember to smile, because smiles are contagious you see. And when you feel down and put on a frown, turn that frown upside down, just for me. Because smiles make us happy and will help us cope with having this dastardly pulmonary hypertension disease. I'm Merle Reeseman and I'm aware that I'm PH rare.

]]>In this episode, pulmonary hypertension patient, Merle Reeseman, discusses her PH diagnosis and the importance of being seen by a PH specialist who understands the disease and can diagnose and prescribe the proper medication.

]]>05:56cleanblood,research,heart,awareness,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseaseIn this episode, pulmonary hypertension patient, Merle Reeseman, discusses her PH diagnosis and the importance of being seen by a PH specialist who understands the disease and can diagnose and prescribe the proper medication.
Learn more about pulmonary hypertension at www.phaware.global Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate @antidote_me @accpchest #phaware 323fullphaware global associationKevin Burke - phaware® interview 221Fri, 15 Feb 2019 13:00:00 +0000

My name is Kevin Burke. I'm from Australia and I'm a pulmonary hypertension patient.

In 2010, I was diagnosed with pulmonary hypertension. I just was getting short of breath, probably like most people do over a number of years. But being a man, never went to the doctor. I was old, unfit, not playing sport anymore and working shift work, so I put all those excuses together. It wasn't until one night at work I thought I was having a stroke. I got sent to hospital. Stroke was ruled out. I was sent to a major center later on for a catheter and discovered that I've got congenital heart defect and pulmonary hypertension at the same time.

Before that, I didn't know I had congenital heart defect or pulmonary hypertension, I just got short of breath. My journey since then has been a rocky one. To start with, I think, probably like most people, I felt isolated and alone, especially living in a rural town in Australia. If you know, Australia's pretty big and we can be isolated quite quickly. A couple of years before I realized there's lots of other people out there with pulmonary hypertension. I found a support group, which is PHAA and it was an amazing journey with them to realize that I have friends who are going through the same, probably mental issues that I was going through as well as the health issues.

Joining the support group and dealing with those mental issues was probably more important and realizing that people go through the same emotions as what I was going through, especially in that initial period. Since joining the group, I ended up being on the general committee for about a year and I'm now the vice president of Pulmonary Hypertension Association Australia. My role, I suppose, is more behind the scenes with trying to lobby government and politicians and drug companies and things like that.

Everybody knows that when you're depressed, you're depressed. You don't have to be told by a professional that you're depressed. So how do I deal with that? Well, obviously, I get on the support group and just say I'm having a low day. In Australia, we have a saying, and it's called "I'm suffering from the black dog." I want to kick the black dog because he's biting me, basically. I deal with it through the support group and the support of many people who go through the same emotions every day. You know that it's going to get better. I live by (I don't if you know the comedian Stephen Fry, who wrote a letter to a lady who was suffering from depression). Read it here. It is a fantastic way to explain how to deal with depression and he talks about peaks and troughs. So, we go through peaks where we're good. Then we go through troughs. Yes, we're going to have bad days but what we've got to remember about those bad days, at the end of those bad days, there's another peak coming, and you'll have good days. And yes, you have good days and you will have another trough coming and have bad days. And it's how you live through those troughs to get to that peak. It’s a very good story.

I also go to counseling. My marriage broke down, sort of made me go to counseling. It was offered to me. It was the best thing I've ever done, and I highly recommend it. A lot of men probably don't go down that avenue or path because of the stigma of men getting that sort of help. I recommend to any man, if it's offered to you, counseling, professional help, take it, grasp it, use it and you'll be better for it because I know I am.

Currently in Australia we have eight medications available. The health system, obviously, in Australia is universal healthcare, a lot different than the United States. One of the things we've been working hard on is getting another drug approved through the pharmaceutical benefits advisory committee so that's to do with getting drugs cheaper and reimbursed.

I went to a meeting last year with Medicines Australia in Canberra and from just a chance meeting with the head of one of the drug companies at a meeting, I was able to facilitate the PBAC to come and speak to us as a patient group. In the whole time of their existence, the PBAC, we're the 13th patient group in the country that they've spoken to in their lifetime. We're the first patient group that they actually come to us. Usually people have to go to Sydney or Canberra to speak to those guys. On our patient day that we have once a year, the PBAC and four delegates come with a heap of information that they wanted to gather from the 35 patients at the patient day. And out of that, they took back a lot of information with them in regard to the needs of pulmonary hypertension patients and what it means to be able to have the availability of more drugs, that obviously are available in other parts of the world. Even doing all of that, they still knocked back the latest drug that we're trying to get approved. So, it's still hard work. And like, I think, most governments, it costs them money and they don't want to spend money on things that they're not going to make any money back on. So that's the hard road for us.

Awareness is the key, isn't it? Everybody needs to be aware. I think the medical community [needs to be aware]. I think what my vision for the future is if somebody presents at their local doctor in their local town, it doesn't matter if there's 100,000 or 1,000 people living in that town and you present with a shortness of breath, all avenues of shortness of breath are investigated. And I think that comes through awareness.

If the medical community are aware that there's not just one avenue of shortness of breath that I think that we're all familiar with, a lot of people getting misdiagnosed. There are other avenues then I think we're on the right path to making sure people get treatment earlier rather than later.

I think we've heard this advice plenty of times before. Don't go on the internet. And I know that was one of the best bits of advice my specialist first gave me. He was adamant. Don't Dr. Google or don't get on the internet and search up because what you read is not reality. There is some truth to what you read, but there's a lot of misinformation out there as well. So, don't get on the internet. Trust your specialist. Listen to everything. If you have questions, write them down in a notepad so next time you see your specialist, you're prepared with a lot of questions instead of trying to go in there and trying to remember everything.

I write a lot of letters that don't get answered to politicians, especially local members. It's crazy. I've got nothing to offer them. I'm asking for something. I'm not giving something. So, engaging a politician is pretty hard. There are things like last year World PH Day in May, I actually hounded the daylights out of my local newspaper with my story. I kept messaging, messaging, messaging and so on the fourth of May, they rang me up and said, "We want to do an interview for the paper." And finally got my page in the paper and the feedback from the community, small community, I suppose, 50,000 people that's small, compared to a lot of cities. The feedback from a lot of people in regard to that was pretty good. We were able to get some awareness raised, through my local paper through persistence.

My name is Kevin Burke. I'm from Australia and I'm a pulmonary hypertension patient.

In 2010, I was diagnosed with pulmonary hypertension. I just was getting short of breath, probably like most people do over a number of years. But being a man, never went to the doctor. I was old, unfit, not playing sport anymore and working shift work, so I put all those excuses together. It wasn't until one night at work I thought I was having a stroke. I got sent to hospital. Stroke was ruled out. I was sent to a major center later on for a catheter and discovered that I've got congenital heart defect and pulmonary hypertension at the same time.

Before that, I didn't know I had congenital heart defect or pulmonary hypertension, I just got short of breath. My journey since then has been a rocky one. To start with, I think, probably like most people, I felt isolated and alone, especially living in a rural town in Australia. If you know, Australia's pretty big and we can be isolated quite quickly. A couple of years before I realized there's lots of other people out there with pulmonary hypertension. I found a support group, which is PHAA and it was an amazing journey with them to realize that I have friends who are going through the same, probably mental issues that I was going through as well as the health issues.

Joining the support group and dealing with those mental issues was probably more important and realizing that people go through the same emotions as what I was going through, especially in that initial period. Since joining the group, I ended up being on the general committee for about a year and I'm now the vice president of Pulmonary Hypertension Association Australia. My role, I suppose, is more behind the scenes with trying to lobby government and politicians and drug companies and things like that.

Everybody knows that when you're depressed, you're depressed. You don't have to be told by a professional that you're depressed. So how do I deal with that? Well, obviously, I get on the support group and just say I'm having a low day. In Australia, we have a saying, and it's called "I'm suffering from the black dog." I want to kick the black dog because he's biting me, basically. I deal with it through the support group and the support of many people who go through the same emotions every day. You know that it's going to get better. I live by (I don't if you know the comedian Stephen Fry, who wrote a letter to a lady who was suffering from depression). Read it here. It is a fantastic way to explain how to deal with depression and he talks about peaks and troughs. So, we go through peaks where we're good. Then we go through troughs. Yes, we're going to have bad days but what we've got to remember about those bad days, at the end of those bad days, there's another peak coming, and you'll have good days. And yes, you have good days and you will have another trough coming and have bad days. And it's how you live through those troughs to get to that peak. It’s a very good story.

I also go to counseling. My marriage broke down, sort of made me go to counseling. It was offered to me. It was the best thing I've ever done, and I highly recommend it. A lot of men probably don't go down that avenue or path because of the stigma of men getting that sort of help. I recommend to any man, if it's offered to you, counseling, professional help, take it, grasp it, use it and you'll be better for it because I know I am.

Currently in Australia we have eight medications available. The health system, obviously, in Australia is universal healthcare, a lot different than the United States. One of the things we've been working hard on is getting another drug approved through the pharmaceutical benefits advisory committee so that's to do with getting drugs cheaper and reimbursed.

I went to a meeting last year with Medicines Australia in Canberra and from just a chance meeting with the head of one of the drug companies at a meeting, I was able to facilitate the PBAC to come and speak to us as a patient group. In the whole time of their existence, the PBAC, we're the 13th patient group in the country that they've spoken to in their lifetime. We're the first patient group that they actually come to us. Usually people have to go to Sydney or Canberra to speak to those guys. On our patient day that we have once a year, the PBAC and four delegates come with a heap of information that they wanted to gather from the 35 patients at the patient day. And out of that, they took back a lot of information with them in regard to the needs of pulmonary hypertension patients and what it means to be able to have the availability of more drugs, that obviously are available in other parts of the world. Even doing all of that, they still knocked back the latest drug that we're trying to get approved. So, it's still hard work. And like, I think, most governments, it costs them money and they don't want to spend money on things that they're not going to make any money back on. So that's the hard road for us.

Awareness is the key, isn't it? Everybody needs to be aware. I think the medical community [needs to be aware]. I think what my vision for the future is if somebody presents at their local doctor in their local town, it doesn't matter if there's 100,000 or 1,000 people living in that town and you present with a shortness of breath, all avenues of shortness of breath are investigated. And I think that comes through awareness.

If the medical community are aware that there's not just one avenue of shortness of breath that I think that we're all familiar with, a lot of people getting misdiagnosed. There are other avenues then I think we're on the right path to making sure people get treatment earlier rather than later.

I think we've heard this advice plenty of times before. Don't go on the internet. And I know that was one of the best bits of advice my specialist first gave me. He was adamant. Don't Dr. Google or don't get on the internet and search up because what you read is not reality. There is some truth to what you read, but there's a lot of misinformation out there as well. So, don't get on the internet. Trust your specialist. Listen to everything. If you have questions, write them down in a notepad so next time you see your specialist, you're prepared with a lot of questions instead of trying to go in there and trying to remember everything.

I write a lot of letters that don't get answered to politicians, especially local members. It's crazy. I've got nothing to offer them. I'm asking for something. I'm not giving something. So, engaging a politician is pretty hard. There are things like last year World PH Day in May, I actually hounded the daylights out of my local newspaper with my story. I kept messaging, messaging, messaging and so on the fourth of May, they rang me up and said, "We want to do an interview for the paper." And finally got my page in the paper and the feedback from the community, small community, I suppose, 50,000 people that's small, compared to a lot of cities. The feedback from a lot of people in regard to that was pretty good. We were able to get some awareness raised, through my local paper through persistence.

Scott Visovatti, MD discusses how early treatment of patients with abnormal exercise studies can delay, or even prevent, the development of pulmonary arterial hypertension. Dr. Visovatti is an Assistant Professor of Internal Medicine in the Division of Cardiology at the University of Michigan. As a physician-scientist in their Pulmonary Hypertension Program, he cares for patients in clinic, performs clinical research that investigates the role of exercise in the diagnosis of pulmonary hypertension, and runs a basic science research lab aimed at exploring the role of purinergic signaling in the development of pulmonary arterial hypertension.

My name is Dr. Scott Visovatti. I am a cardiologist and assistant professor of internal medicine at the University of Michigan. I'd like to talk a little bit about the role of exercise in the diagnosis, and perhaps even, predicting the future of patients with pulmonary hypertension.

What we know is that there are groups of folks who are at risk for developing pulmonary hypertension, or pulmonary arterial hypertension, which is a sub group. Those folks include patients with connective tissue disease, like scleroderma, or patients that have had blood clots in their lungs. Now we know not everyone with scleroderma, or everyone with a blood clot in their lung will go on to develop high pressures in their lung, which can ultimately lead to right sided heart failure and death. Obviously, this is a critical issue to figure out.

How do we tell a patient what the future may hold?

If I could give an example of a patient with scleroderma who comes in to see their rheumatologist, or their cardiologist, or their pulmonologist, and that person is concerned that their exercise status has changed. They can't do as much as they could at last visit. They're having difficulty climbing a flight of stairs. Or even speaking complete sentences. Typically, we would get an echocardiogram, perhaps pulmonary function tests, other studies, that would accurately assess what the pressures are in the heart and in the lungs. That's something that we do with a study called the right heart catheterization. [A right heart cath} is basically putting an IV in the side of the neck, or in the groin, into a vein, and threading a catheter that can measure pressure up through the heart and into the main artery of the lung. That's the gold standard test. There are specific cut offs that tell us whether or not a patient has pulmonary arterial hypertension, based on pressures and some calculations.

The difficult situation here is that sometimes we find that they don't meet criteria for pulmonary arterial hypertension, yet they're still feeling poorly and there's always that cloud hanging over them in the sense that great, today they don't meet the diagnosis criteria, but what does the next week, or month, or even year hold in store for them?

Based on some work that we've done at the University of California, Los Angeles, by Dr. Khanna, who's a very prominent rheumatologist who we're happy to have here at Michigan, and we work very closely together. He found that exercise can basically unmask different profiles. Meaning different collections of pressure data that might identify folks who are at higher risk for the development of pulmonary arterial hypertension down the line.

We're working with him and Dr. McLaughlin and the group here at Michigan. We started not only performing these right heart catheterizations, measuring pressures at rest, but also with exercise. It sounds a little bizarre, but we can slide a bicycle, what we call an ergometer on the end of the table, where the patient is undergoing the heart catheterization. They can exercise while that catheter is in place. What we found is that there are very distinct patterns that can emerge when folks are really pushing themselves. The exciting thing here is that we are finding sort of a middle ground between normal pressures and pressures that would be diagnostic of pulmonary arterial hypertension. We think that this middle group, this pattern, that sort of in between those two extremes, it might predict somebody who is more likely to go on to develop this horrible disease.

If we can identify those folks, we can follow them more closely. Our hope is that we can actually begin clinical trials to see if treating people with medication at that earlier intermediate stage would be helpful in preventing the actual development down the line of this disease. But that's something that we're working on very carefully right now. It's a hot topic and it really deserves some careful attention here.

What came out of the newest World Symposium is this idea that our strict cut off for what's an abnormal pressure in the pulmonary artery was perhaps a little higher than it should be. In other words, from studies that show what a normal pressure is, we know that there's sort of a range of normal and abnormal. So, what our experts in Nice found last year was that we need to perhaps lower that cut off. So the mean pulmonary arterial pressure cut off had been 25 millimeters of mercury. Meaning up until that point, that was considered to be normal, or high normal. The important thing here is that the definition also required some calculations that showed that the arteries in the lungs were clamped down about three times normal, or greater. The new definition lowers that mean pulmonary arterial pressure threshold to 20 millimeters of mercury. So, a lower cut off there potentially capturing more people. However, that important point is that pulmonary vascular resistance, or the degree to which the arteries are clamped down, it's still necessary that that calculation shows three times normal, or greater.

We've gone back since this new definition was put forth, and looked at our past diagnosis and found that this changed categories for very few people. Meaning that that would be a very unusual person that would now be included in the group of pulmonary arterial hypertension. But for that one person, that's a critical difference. That's somebody that we could now treat. Whereas before, it would be more difficult to justify treatment. So it's a slight difference. It doesn't really change the exercise world. What that last World Symposium concluded is that we still really don't have enough solid clinical data. Enough clinical trials to justify having a consensus definition for what the role of exercise in pulmonary arterial hypertension [is]. Which is why a lot of us who are working in this field are doubling our efforts to try to show that we do think exercise can show the middle ground that is on the spectrum. Meaning from health to disease.

What we think is also exciting here is we're growing the offerings that we have for folks with blood clots in their lungs. Something called pulmonary embolism, which is a blood clot that typically breaks off from the legs and goes to the lungs. For most people that is something that the body handles by breaking down that blood clot. For a small group of people, the body doesn't have that capability or lacks the potency necessary to completely break down all those clots. So over time those blood clots can actually turn into scar tissue. What our center and others are offering is either a type of open heart surgery to surgically remove that scar tissue, or in a group of patients who aren't candidates for surgery, we are offering something called balloon pulmonary angioplasty, which is a little bit like what we offer to patients who have blockages in their coronary artery. We can put a catheter in and use a balloon to move that scar tissue off to the side and help blood flow to the lungs.

But like that patient with scleroderma, we first have to prove that those pressures are high enough to give that person a diagnosis of what's called chronic thromboembolic pulmonary hypertension [CTEPH]. There are strict cut offs in terms of pressures. What we are finding is that we'll take a patient to the cath lab, perform that right heart catheterization and find that those pressures don't quite meet that definition of pulmonary hypertension.

Again, they're feeling poorly, they're short of breath, they can't do what they did before they had this pulmonary embolism. So how do we help them? Well, we're using exercise here as well. Meaning all too often, it's the case that climbing a flight of stairs or going up an incline is the thing that's most difficult for patients like this. We want to try to recreate for their body, that same stress by exercising them, and watching to see if patterns of pressures show that sort of intermediate group, who are not quite normal, but still don't meet that definition of pulmonary hypertension.

We believe that this may be a group who may require something sooner, rather than later. That something could be medication(s). It could be an angioplasty. Perhaps it's somebody who we need to watch all the more closely to see if surgery is in their future. So once again, using exercise as an evocative maneuver. A way to kind of put the body under a stressful situation to see if that can help us identify folks who really do need to be intervened upon earlier, rather than later.

Scott Visovatti, MD discusses how early treatment of patients with abnormal exercise studies can delay, or even prevent, the development of pulmonary arterial hypertension. Dr. Visovatti is an Assistant Professor of Internal Medicine in the Division of Cardiology at the University of Michigan. As a physician-scientist in their Pulmonary Hypertension Program, he cares for patients in clinic, performs clinical research that investigates the role of exercise in the diagnosis of pulmonary hypertension, and runs a basic science research lab aimed at exploring the role of purinergic signaling in the development of pulmonary arterial hypertension.

My name is Dr. Scott Visovatti. I am a cardiologist and assistant professor of internal medicine at the University of Michigan. I'd like to talk a little bit about the role of exercise in the diagnosis, and perhaps even, predicting the future of patients with pulmonary hypertension.

What we know is that there are groups of folks who are at risk for developing pulmonary hypertension, or pulmonary arterial hypertension, which is a sub group. Those folks include patients with connective tissue disease, like scleroderma, or patients that have had blood clots in their lungs. Now we know not everyone with scleroderma, or everyone with a blood clot in their lung will go on to develop high pressures in their lung, which can ultimately lead to right sided heart failure and death. Obviously, this is a critical issue to figure out.

How do we tell a patient what the future may hold?

If I could give an example of a patient with scleroderma who comes in to see their rheumatologist, or their cardiologist, or their pulmonologist, and that person is concerned that their exercise status has changed. They can't do as much as they could at last visit. They're having difficulty climbing a flight of stairs. Or even speaking complete sentences. Typically, we would get an echocardiogram, perhaps pulmonary function tests, other studies, that would accurately assess what the pressures are in the heart and in the lungs. That's something that we do with a study called the right heart catheterization. [A right heart cath} is basically putting an IV in the side of the neck, or in the groin, into a vein, and threading a catheter that can measure pressure up through the heart and into the main artery of the lung. That's the gold standard test. There are specific cut offs that tell us whether or not a patient has pulmonary arterial hypertension, based on pressures and some calculations.

The difficult situation here is that sometimes we find that they don't meet criteria for pulmonary arterial hypertension, yet they're still feeling poorly and there's always that cloud hanging over them in the sense that great, today they don't meet the diagnosis criteria, but what does the next week, or month, or even year hold in store for them?

Based on some work that we've done at the University of California, Los Angeles, by Dr. Khanna, who's a very prominent rheumatologist who we're happy to have here at Michigan, and we work very closely together. He found that exercise can basically unmask different profiles. Meaning different collections of pressure data that might identify folks who are at higher risk for the development of pulmonary arterial hypertension down the line.

We're working with him and Dr. McLaughlin and the group here at Michigan. We started not only performing these right heart catheterizations, measuring pressures at rest, but also with exercise. It sounds a little bizarre, but we can slide a bicycle, what we call an ergometer on the end of the table, where the patient is undergoing the heart catheterization. They can exercise while that catheter is in place. What we found is that there are very distinct patterns that can emerge when folks are really pushing themselves. The exciting thing here is that we are finding sort of a middle ground between normal pressures and pressures that would be diagnostic of pulmonary arterial hypertension. We think that this middle group, this pattern, that sort of in between those two extremes, it might predict somebody who is more likely to go on to develop this horrible disease.

If we can identify those folks, we can follow them more closely. Our hope is that we can actually begin clinical trials to see if treating people with medication at that earlier intermediate stage would be helpful in preventing the actual development down the line of this disease. But that's something that we're working on very carefully right now. It's a hot topic and it really deserves some careful attention here.

What came out of the newest World Symposium is this idea that our strict cut off for what's an abnormal pressure in the pulmonary artery was perhaps a little higher than it should be. In other words, from studies that show what a normal pressure is, we know that there's sort of a range of normal and abnormal. So, what our experts in Nice found last year was that we need to perhaps lower that cut off. So the mean pulmonary arterial pressure cut off had been 25 millimeters of mercury. Meaning up until that point, that was considered to be normal, or high normal. The important thing here is that the definition also required some calculations that showed that the arteries in the lungs were clamped down about three times normal, or greater. The new definition lowers that mean pulmonary arterial pressure threshold to 20 millimeters of mercury. So, a lower cut off there potentially capturing more people. However, that important point is that pulmonary vascular resistance, or the degree to which the arteries are clamped down, it's still necessary that that calculation shows three times normal, or greater.

We've gone back since this new definition was put forth, and looked at our past diagnosis and found that this changed categories for very few people. Meaning that that would be a very unusual person that would now be included in the group of pulmonary arterial hypertension. But for that one person, that's a critical difference. That's somebody that we could now treat. Whereas before, it would be more difficult to justify treatment. So it's a slight difference. It doesn't really change the exercise world. What that last World Symposium concluded is that we still really don't have enough solid clinical data. Enough clinical trials to justify having a consensus definition for what the role of exercise in pulmonary arterial hypertension [is]. Which is why a lot of us who are working in this field are doubling our efforts to try to show that we do think exercise can show the middle ground that is on the spectrum. Meaning from health to disease.

What we think is also exciting here is we're growing the offerings that we have for folks with blood clots in their lungs. Something called pulmonary embolism, which is a blood clot that typically breaks off from the legs and goes to the lungs. For most people that is something that the body handles by breaking down that blood clot. For a small group of people, the body doesn't have that capability or lacks the potency necessary to completely break down all those clots. So over time those blood clots can actually turn into scar tissue. What our center and others are offering is either a type of open heart surgery to surgically remove that scar tissue, or in a group of patients who aren't candidates for surgery, we are offering something called balloon pulmonary angioplasty, which is a little bit like what we offer to patients who have blockages in their coronary artery. We can put a catheter in and use a balloon to move that scar tissue off to the side and help blood flow to the lungs.

But like that patient with scleroderma, we first have to prove that those pressures are high enough to give that person a diagnosis of what's called chronic thromboembolic pulmonary hypertension [CTEPH]. There are strict cut offs in terms of pressures. What we are finding is that we'll take a patient to the cath lab, perform that right heart catheterization and find that those pressures don't quite meet that definition of pulmonary hypertension.

Again, they're feeling poorly, they're short of breath, they can't do what they did before they had this pulmonary embolism. So how do we help them? Well, we're using exercise here as well. Meaning all too often, it's the case that climbing a flight of stairs or going up an incline is the thing that's most difficult for patients like this. We want to try to recreate for their body, that same stress by exercising them, and watching to see if patterns of pressures show that sort of intermediate group, who are not quite normal, but still don't meet that definition of pulmonary hypertension.

We believe that this may be a group who may require something sooner, rather than later. That something could be medication(s). It could be an angioplasty. Perhaps it's somebody who we need to watch all the more closely to see if surgery is in their future. So once again, using exercise as an evocative maneuver. A way to kind of put the body under a stressful situation to see if that can help us identify folks who really do need to be intervened upon earlier, rather than later.

]]>fullEpisode 220 - Scott Visovatti, MDScott Visovatti, MD - Pulmonary HypertensionMon, 11 Feb 2019 13:00:00 +0000In this episode, Scott Visovatti, MD discusses how early treatment of patients with abnormal exercise studies can delay, or even prevent, the development of pulmonary arterial hypertension. Dr. Visovatti is an Assistant Professor of Internal Medicine in the Division of Cardiology at the University of Michigan. As a physician-scientist in their Pulmonary Hypertension Program, he cares for patients in clinic, performs clinical research that investigates the role of exercise in the diagnosis of pulmonary hypertension, and runs a basic science research lab aimed at exploring the role of purinergic signaling in the development of pulmonary arterial hypertension.

]]>In this episode, Scott Visovatti, MD discusses how early treatment of patients with abnormal exercise studies can delay, or even prevent, the development of pulmonary arterial hypertension. Dr. Visovatti is an Assistant Professor of Internal Medicine in the Division of Cardiology at the University of Michigan. As a physician-scientist in their Pulmonary Hypertension Program, he cares for patients in clinic, performs clinical research that investigates the role of exercise in the diagnosis of pulmonary hypertension, and runs a basic science research lab aimed at exploring the role of purinergic signaling in the development of pulmonary arterial hypertension.

]]>09:18cleanblood,research,heart,awareness,therapies,asthma,breathless,rare,ph,trials,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdisease,clincalIn this episode, Scott Visovatti, MD discusses how early treatment of patients with abnormal exercise studies can delay, or even prevent, the development of pulmonary arterial hypertension. Dr. Visovatti is an Assistant Professor of Internal Medicine in the Division of Cardiology at the University of Michigan.
Learn more about pulmonary hypertension at www.phaware.global @phaware @umichmedicine @umichCVC @antidote_me @accpchest #phaware #phawareMD321fullphaware global associationValerie McLaughlin, MD - phaware® interview 219Fri, 08 Feb 2019 13:00:00 +0000

Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center discusses what a pulmonary hypertension patients should expect in theirfirst visit to an expert PH Center. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field.

Hi. I'm Vallerie McLaughlin from the University of Michigan. I direct the pulmonary hypertension program there and today it's my pleasure to talk to you a little bit about what to expect once you get to an expert [pulmonary hypertension] center.

Most patients get referred to us often after having seen a number of physicians for shortness of breath, often with an echocardiogram that shows an elevated pulmonary artery pressure. Now, one thing that's really important to remember is that the echocardiogram is just an estimate of pulmonary artery pressure and there's much more to an echo than just the pulmonary artery pressure. We look at the size and function of the right ventricle.

It's very often that the symptoms of pulmonary hypertension are nonspecific and that means those symptoms could occur in many different diseases; shortness of breath, fatigue, chest discomfort and the like. It's important for us to look for other causes of such symptoms; things like lung disease, COPD, and pulmonary fibrosis. Oftentimes a patient get testing on their lungs like pulmonary function tests or a chest CT scan or a study for obstructive sleep apnea. That's really important to look for.

Sometimes patients have other heart problems; their left heart, their ventricles don't squeeze well or don't relax well or there's a problem with the valves on the left side of the heart. That's actually a very common cause of pulmonary hypertension. Sometimes patients have blood clots in the lungs that cause pulmonary hypertension and so doing a ventilation/perfusion scan to rule that out is important.

After we've looked for all of these other causes, and there are many more besides the ones I mentioned, we want to confirm a diagnosis of pulmonary hypertension. That requires a right heart catheterization. A right heart catheterization is a study that is done usually in a cardiac catheterization lab during which we put a small catheter in a blood vessel, often in the neck or in the groin area. We thread that catheter to the heart and we measure the pressure in the different chambers of the heart and lungs and how the blood flow goes through the lungs.

Sometimes when we do this, we learn that the problem is on the left side of the heart, that the patient has pulmonary hypertension because the left heart is sending too much pressure back to the lungs. Sometimes we learn that the patient has what we call pulmonary arterial hypertension. PAH is high pressure in the lungs with an elevated pressure and elevated resistance across that vascular bed in the lungs and no other heart or lung disease. That can be idiopathic. It can be hereditary, or it can be due to some other diseases such as connective tissue diseases like scleroderma or lupus. Occasionally, it's due to liver disease. Sometimes it's due to holes in the heart. Occasionally, it's due to different drugs and toxins like diet pills or methamphetamines. But the right heart catheterization is absolutely required for the diagnosis.

Oftentimes, we'll try to get an assessment of how sick a patient is; what their risk level is. Not just from the numbers we measure on the catheterization, but also things like BNP, a blood test to determine stress on the heart, and how far the patient can walk in some sort of exercise tolerance test, often a six-minute hall walk. So once the diagnosis is complete, let's say that we have ruled out the other heart diseases that can do this, the other lung diseases that can do this, and we're calling the patient Group I pulmonary arterial hypertension; we decide what treatment is best for them based on their risk status and again, that involves a number of different factors.

There are medications that generally belong to three different groups that are now FDA approved to treat pulmonary hypertension. The prostacyclin pathway and medications in this pathway come in oral, inhaled and subcutaneous (under the skin), and IV into the vein forms. There is the nitric oxide pathway. In this pathway, are the PDE5 inhibitors and the sGC stimulators. These are oral therapies. {Also, there's} the endothelin receptor pathway. These are oral therapies, as well.

In the very sickest of the sick patients, the patients who come to us with very advanced symptoms who have heart catheterization numbers signifying advanced disease with a high right atrial pressure and a low cardiac index, we often go straight to the parenteral prostacyclin therapies. {These are] the IV therapies and the under-the-skin therapies, often in combination with oral agents, as well. We feel those patients are at such high risk that we really need to be very aggressive with their therapy. In our experience at the University of Michigan, I would say that's probably about 10% of the new PAH patients that we see. So it's not the majority, but there are certainly patients that fall into that category.

I would say the majority of newly diagnosed patients that we see now start on dual oral therapy and that's usually the combination of an endothelin receptor antagonist and a PDE5 inhibitor based on clinical trials. It's very remarkable the improvement that patients have with up-front oral combination therapy compared to just one medication at a time. I would say that has now become the standard when we see patients that are newly diagnosed who are in either the low or the moderate risk range. There are some opportunities to use just one medication and those are few and far between. In fact, my approach now is there needs to be a really good reason to start with only one medication as opposed to use two at the time of diagnosis.

This first treatment [information] is really just that, just the first step. After you make the first treatment decision, it's really important to continue to monitor the patient to see how they do and continue to adjust their medications to try to get them into that very lowest risk status.

This is Vallerie McLaughlin from the University of Michigan and that's a little summary of what might happen to a patient after they've been referred to a pulmonary hypertension center.

Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center discusses what a pulmonary hypertension patients should expect in theirfirst visit to an expert PH Center. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Hi. I'm Vallerie McLaughlin from the University of Michigan. I direct the pulmonary hypertension program there and today it's my pleasure to talk to you a little bit about what to expect once you get to an expert [pulmonary hypertension] center.

Most patients get referred to us often after having seen a number of physicians for shortness of breath, often with an echocardiogram that shows an elevated pulmonary artery pressure. Now, one thing that's really important to remember is that the echocardiogram is just an estimate of pulmonary artery pressure and there's much more to an echo than just the pulmonary artery pressure. We look at the size and function of the right ventricle.

It's very often that the symptoms of pulmonary hypertension are nonspecific and that means those symptoms could occur in many different diseases; shortness of breath, fatigue, chest discomfort and the like. It's important for us to look for other causes of such symptoms; things like lung disease, COPD, and pulmonary fibrosis. Oftentimes a patient get testing on their lungs like pulmonary function tests or a chest CT scan or a study for obstructive sleep apnea. That's really important to look for.

Sometimes patients have other heart problems; their left heart, their ventricles don't squeeze well or don't relax well or there's a problem with the valves on the left side of the heart. That's actually a very common cause of pulmonary hypertension. Sometimes patients have blood clots in the lungs that cause pulmonary hypertension and so doing a ventilation/perfusion scan to rule that out is important.

After we've looked for all of these other causes, and there are many more besides the ones I mentioned, we want to confirm a diagnosis of pulmonary hypertension. That requires a right heart catheterization. A right heart catheterization is a study that is done usually in a cardiac catheterization lab during which we put a small catheter in a blood vessel, often in the neck or in the groin area. We thread that catheter to the heart and we measure the pressure in the different chambers of the heart and lungs and how the blood flow goes through the lungs.

Sometimes when we do this, we learn that the problem is on the left side of the heart, that the patient has pulmonary hypertension because the left heart is sending too much pressure back to the lungs. Sometimes we learn that the patient has what we call pulmonary arterial hypertension. PAH is high pressure in the lungs with an elevated pressure and elevated resistance across that vascular bed in the lungs and no other heart or lung disease. That can be idiopathic. It can be hereditary, or it can be due to some other diseases such as connective tissue diseases like scleroderma or lupus. Occasionally, it's due to liver disease. Sometimes it's due to holes in the heart. Occasionally, it's due to different drugs and toxins like diet pills or methamphetamines. But the right heart catheterization is absolutely required for the diagnosis.

Oftentimes, we'll try to get an assessment of how sick a patient is; what their risk level is. Not just from the numbers we measure on the catheterization, but also things like BNP, a blood test to determine stress on the heart, and how far the patient can walk in some sort of exercise tolerance test, often a six-minute hall walk. So once the diagnosis is complete, let's say that we have ruled out the other heart diseases that can do this, the other lung diseases that can do this, and we're calling the patient Group I pulmonary arterial hypertension; we decide what treatment is best for them based on their risk status and again, that involves a number of different factors.

There are medications that generally belong to three different groups that are now FDA approved to treat pulmonary hypertension. The prostacyclin pathway and medications in this pathway come in oral, inhaled and subcutaneous (under the skin), and IV into the vein forms. There is the nitric oxide pathway. In this pathway, are the PDE5 inhibitors and the sGC stimulators. These are oral therapies. {Also, there's} the endothelin receptor pathway. These are oral therapies, as well.

In the very sickest of the sick patients, the patients who come to us with very advanced symptoms who have heart catheterization numbers signifying advanced disease with a high right atrial pressure and a low cardiac index, we often go straight to the parenteral prostacyclin therapies. {These are] the IV therapies and the under-the-skin therapies, often in combination with oral agents, as well. We feel those patients are at such high risk that we really need to be very aggressive with their therapy. In our experience at the University of Michigan, I would say that's probably about 10% of the new PAH patients that we see. So it's not the majority, but there are certainly patients that fall into that category.

I would say the majority of newly diagnosed patients that we see now start on dual oral therapy and that's usually the combination of an endothelin receptor antagonist and a PDE5 inhibitor based on clinical trials. It's very remarkable the improvement that patients have with up-front oral combination therapy compared to just one medication at a time. I would say that has now become the standard when we see patients that are newly diagnosed who are in either the low or the moderate risk range. There are some opportunities to use just one medication and those are few and far between. In fact, my approach now is there needs to be a really good reason to start with only one medication as opposed to use two at the time of diagnosis.

This first treatment [information] is really just that, just the first step. After you make the first treatment decision, it's really important to continue to monitor the patient to see how they do and continue to adjust their medications to try to get them into that very lowest risk status.

This is Vallerie McLaughlin from the University of Michigan and that's a little summary of what might happen to a patient after they've been referred to a pulmonary hypertension center.

]]>fullEpisode 219 - Vallerie McLaughlin, MDVallerie McLaughlin, MD - Expert Pulmonary Hypertension CentersThu, 07 Feb 2019 13:00:00 +0000In this episode, Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center discusses what a pulmonary hypertension patients should expect in their first visit to an expert PH Center. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field.

]]>In this episode, Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center discusses what a pulmonary hypertension patients should expect in their first visit to an expert PH Center. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field.

]]>06:30cleanblood,research,heart,awareness,therapies,asthma,breathless,rare,ph,trials,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdisease,clincalIn this episode, Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center discusses what a pulmonary hypertension patients should expect in their first visit to an expert PH Center. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor.
Learn more about pulmonary hypertension at www.phaware.global @phaware @umichmedicine @umichCVC @antidote_me @accpchest #phaware #phawareMD320fullphaware global association Valerie McLaughlin, MD - phaware® interview 218Tue, 05 Feb 2019 13:00:00 +0000

Vallerie McLaughlin, MD gives an overview of6th World Symposium on Pulmonary Hypertension, which took place in Nice, France, in early 2018. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field.

My name is Valerie McLaughlin, I'm a cardiologist at the University of Michigan, and my area of expertise is pulmonary hypertension. I've been doing this for over 20 years and it's been a really remarkable ride.

It's very exciting today to talk to you about some of the latest discussions that occurred at the 6th World Symposium on Pulmonary Hypertension, which took place in Nice, France, in early 2018.

I had the pleasure of attending the symposium and being involved in one of the task forces, and I attended all of the presentations of the task forces. There were 13 task forces at the World Symposium, and it's really a great opportunity for experts in the field to review the literature, make recommendations, not only about how patients should be treated now, but about what things are likely to make an impact in the future.

So, it's important that we secure funding for this. That's probably step one. But then we put together the task force years in advance, and they start working well in advance of the actual meeting. In fact, hopefully, they come to the meeting with a number of documents in place, so that they can talk as a group about what the best recommendations are. After they do that, the recommendations for each of the task forces are presented to the whole population, all of the attendees, and then there is an opportunity for discussion.

After that discussion, we then take into account all of the discussion, all of the perspectives, and then write a manuscript. Those manuscripts get published as a supplement or as a journal, and in this case, it's now published in the European Respiratory Journal.

The first thing that really changed at the World Symposium was the definition of pulmonary hypertension. As you all probably know, pulmonary hypertension is high blood pressure in the lungs. Just like there is a blood pressure in the arm, there is a blood pressure in the lungs too. And sometimes when the blood pressure in the lungs gets elevated, the right side of the heart has a hard time pushing the blood forward, and the patient becomes symptomatic with the most common symptom being shortness of breath.

Many years ago, the cut-off for the definition of pulmonary hypertension was defined as a mean pulmonary artery pressure of greater than 25. That was really decided quite arbitrarily at a conference many, many years ago, but that definition has been carried forward. Over the more recent years, we've learned from studies doing heart catheterizations on patients, including patients who don't have the disease, that the cut-off of 25 may be too high. In fact, the normal population should have a cut-off of no more than 20 for their mean pulmonary pressure. Now that's not the top number, that's the mean pulmonary artery pressure. And this was an area that was discussed in some detail at the World Symposium. After reviewing the data, the definition has now changed.

So [now] to be called pulmonary arterial hypertension, one has to have a mean pulmonary artery pressure of greater than 20, measured at right heart catheterization, with a wedge pressure (or left heart filling pressure) of less than 15 and a calculated pulmonary vascular resistance of greater than 3 Wood units. The pulmonary vascular resistance is a calculated number that takes into account not only the pressure in the pulmonary arteries, but also how the blood flow goes through the heart. So this is the new hemodynamic definition of pulmonary hypertension.

Another important topic that was discussed at the World Symposium was how to treat pulmonary hypertension and what treatment goals should be. We've learned over the years that there are certain factors that can predict how well a patient is going to do. How to assess the risk can be done in multiple different ways. At the World Symposium, we reviewed a number of ways to assess risk, including the REVEAL risk score, the ESC/ERS recommendations (European Society of Cardiology (ESC) and the European Respiratory Society), and a French approach, which is a more simple approach based on how many low risk factors a patient has.

All of these techniques have many things in common; the things that we know predict a better prognosis. Those things include simply how a patient is doing as measured as functional class. The less symptomatic a patient is, the better they do. There are also measures of exercise tolerance; how far a patient walks on a 6 minute hall walk, or how well they exercise on cardio-pulmonary exercise testing. Their symptoms of heart failure, whether or not they have fluid retention. Some hemodynamic markers, like the cardiac output and right atrial pressure. Biomarkers, a simple blood test like BNP. All of these things are important in predicting risk, and of these 3 different techniques, while they measure different things they all have definitions of what a low risk state is.

The data now show that no matter which technique you use to assess risk, if you can get a patient into the low risk status, they're going to do well. So the new treatment algorithm really emphasizes the importance of risk assessment. If you assess a patient at the time of their diagnosis, one needs to start therapy based on how sick they are. Sometimes patients get to us and they're still very, very sick, they're very advanced, and we need to be very aggressive with therapy, and we might start in fact 3 drugs at once including a prostacyclin like an IV or a SubQ therapy. Sometimes they're not quite as sick and we'll start one, or most often two oral therapies.

But really the key in the new treatment algorithm is the reassessment. No matter what you started a patient on, you have to reassess the patient and see if they're at low risk. If they're at low risk after that first treatment choice, that's great. We just continue to follow them and make sure they stay in that low risk category. If they are not meeting the criteria for low risk after the initial treatment choice, one needs to do something differently, whether that's add another medication or whether that's switch a medication, go on to a more aggressive type of a prostacyclin for example. You need to do something differently. They whole goal of the new treatment algorithm is getting the patient into a low risk status.

At the 6th World Symposium, this was the first time that we had a whole task force dedicated to patients' perspective. We'd always been very physician and researcher oriented in the past, and this year we had a task force that included physicians, nurses, patients, patient advocates, patient family members, and representatives from some of the patient organizations, the associations. Their key was really to talk about different things that are important to patients, and they came out with some very important comments that I think we as a medical community need to take into consideration as we plan subsequent meetings, as we take care of patients, and as we plan clinical research trials.

One thing that they discussed in some detail was quality of life. This is something that's very important, and is a little bit difficult at times for scientists to quantitate. When we do clinical trials to get medications approved, we want something very hard. For example, 6 minute hall walk in the earlier trials, or morbidity and mortality in the later trials. While that is a path to FDA approval of agents, the patients want something that really reflects in their everyday life. And so quality of life measures is something that I think we need to consider more strongly as we do further clinical trials. And in fact this year, there was a new quality of life measurement tool called the PAH-SYMPACT. It's simply a quality of life questionnaire, but it was developed with FDA guidance. I think that will be a nice tool to use in subsequent clinical trials.

Another thing that was discussed in the patient perspective task force, was the concept of rolls of the different participants. This is not just a decision that a physician makes, or another practitioner, it's important to emphasize the concept of what we call shared decision making. The patient and the patient's family members have to play an important role as we treat patients. Some of these therapies that we use are quite complex and it requires a huge commitment by the patient and their family member. And they really need to understand the facts of those therapies, the implications of those therapies, and have to balance whether or not those therapies are right for them.

Another point that was made, something that I as a physician struggle with, is the concept of palliative care. We heard from our patients in the patient perspective that they want to know at palliative care. They want to know those options. For me personally, as a physician, I always feel like the patients come to us for hope, the patients come to us to get better, and it takes a lot for me to kind of say, maybe we're not doing as well as we would like and maybe we need to consider alternative options. But what we heard from the patients is they want to understand those other options sooner rather than later. And so I think that it is important to the medical community and the patients, as well, to be proactive about making sure we discuss all options of patient care, and sometimes that includes palliative care.

Thank you for joining me, I'm Valerie McLaughlin from the University of Michigan, and I'm aware that I'm rare.

Vallerie McLaughlin, MD gives an overview of6th World Symposium on Pulmonary Hypertension, which took place in Nice, France, in early 2018. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field.

My name is Valerie McLaughlin, I'm a cardiologist at the University of Michigan, and my area of expertise is pulmonary hypertension. I've been doing this for over 20 years and it's been a really remarkable ride.

It's very exciting today to talk to you about some of the latest discussions that occurred at the 6th World Symposium on Pulmonary Hypertension, which took place in Nice, France, in early 2018.

I had the pleasure of attending the symposium and being involved in one of the task forces, and I attended all of the presentations of the task forces. There were 13 task forces at the World Symposium, and it's really a great opportunity for experts in the field to review the literature, make recommendations, not only about how patients should be treated now, but about what things are likely to make an impact in the future.

So, it's important that we secure funding for this. That's probably step one. But then we put together the task force years in advance, and they start working well in advance of the actual meeting. In fact, hopefully, they come to the meeting with a number of documents in place, so that they can talk as a group about what the best recommendations are. After they do that, the recommendations for each of the task forces are presented to the whole population, all of the attendees, and then there is an opportunity for discussion.

After that discussion, we then take into account all of the discussion, all of the perspectives, and then write a manuscript. Those manuscripts get published as a supplement or as a journal, and in this case, it's now published in the European Respiratory Journal.

The first thing that really changed at the World Symposium was the definition of pulmonary hypertension. As you all probably know, pulmonary hypertension is high blood pressure in the lungs. Just like there is a blood pressure in the arm, there is a blood pressure in the lungs too. And sometimes when the blood pressure in the lungs gets elevated, the right side of the heart has a hard time pushing the blood forward, and the patient becomes symptomatic with the most common symptom being shortness of breath.

Many years ago, the cut-off for the definition of pulmonary hypertension was defined as a mean pulmonary artery pressure of greater than 25. That was really decided quite arbitrarily at a conference many, many years ago, but that definition has been carried forward. Over the more recent years, we've learned from studies doing heart catheterizations on patients, including patients who don't have the disease, that the cut-off of 25 may be too high. In fact, the normal population should have a cut-off of no more than 20 for their mean pulmonary pressure. Now that's not the top number, that's the mean pulmonary artery pressure. And this was an area that was discussed in some detail at the World Symposium. After reviewing the data, the definition has now changed.

So [now] to be called pulmonary arterial hypertension, one has to have a mean pulmonary artery pressure of greater than 20, measured at right heart catheterization, with a wedge pressure (or left heart filling pressure) of less than 15 and a calculated pulmonary vascular resistance of greater than 3 Wood units. The pulmonary vascular resistance is a calculated number that takes into account not only the pressure in the pulmonary arteries, but also how the blood flow goes through the heart. So this is the new hemodynamic definition of pulmonary hypertension.

Another important topic that was discussed at the World Symposium was how to treat pulmonary hypertension and what treatment goals should be. We've learned over the years that there are certain factors that can predict how well a patient is going to do. How to assess the risk can be done in multiple different ways. At the World Symposium, we reviewed a number of ways to assess risk, including the REVEAL risk score, the ESC/ERS recommendations (European Society of Cardiology (ESC) and the European Respiratory Society), and a French approach, which is a more simple approach based on how many low risk factors a patient has.

All of these techniques have many things in common; the things that we know predict a better prognosis. Those things include simply how a patient is doing as measured as functional class. The less symptomatic a patient is, the better they do. There are also measures of exercise tolerance; how far a patient walks on a 6 minute hall walk, or how well they exercise on cardio-pulmonary exercise testing. Their symptoms of heart failure, whether or not they have fluid retention. Some hemodynamic markers, like the cardiac output and right atrial pressure. Biomarkers, a simple blood test like BNP. All of these things are important in predicting risk, and of these 3 different techniques, while they measure different things they all have definitions of what a low risk state is.

The data now show that no matter which technique you use to assess risk, if you can get a patient into the low risk status, they're going to do well. So the new treatment algorithm really emphasizes the importance of risk assessment. If you assess a patient at the time of their diagnosis, one needs to start therapy based on how sick they are. Sometimes patients get to us and they're still very, very sick, they're very advanced, and we need to be very aggressive with therapy, and we might start in fact 3 drugs at once including a prostacyclin like an IV or a SubQ therapy. Sometimes they're not quite as sick and we'll start one, or most often two oral therapies.

But really the key in the new treatment algorithm is the reassessment. No matter what you started a patient on, you have to reassess the patient and see if they're at low risk. If they're at low risk after that first treatment choice, that's great. We just continue to follow them and make sure they stay in that low risk category. If they are not meeting the criteria for low risk after the initial treatment choice, one needs to do something differently, whether that's add another medication or whether that's switch a medication, go on to a more aggressive type of a prostacyclin for example. You need to do something differently. They whole goal of the new treatment algorithm is getting the patient into a low risk status.

At the 6th World Symposium, this was the first time that we had a whole task force dedicated to patients' perspective. We'd always been very physician and researcher oriented in the past, and this year we had a task force that included physicians, nurses, patients, patient advocates, patient family members, and representatives from some of the patient organizations, the associations. Their key was really to talk about different things that are important to patients, and they came out with some very important comments that I think we as a medical community need to take into consideration as we plan subsequent meetings, as we take care of patients, and as we plan clinical research trials.

One thing that they discussed in some detail was quality of life. This is something that's very important, and is a little bit difficult at times for scientists to quantitate. When we do clinical trials to get medications approved, we want something very hard. For example, 6 minute hall walk in the earlier trials, or morbidity and mortality in the later trials. While that is a path to FDA approval of agents, the patients want something that really reflects in their everyday life. And so quality of life measures is something that I think we need to consider more strongly as we do further clinical trials. And in fact this year, there was a new quality of life measurement tool called the PAH-SYMPACT. It's simply a quality of life questionnaire, but it was developed with FDA guidance. I think that will be a nice tool to use in subsequent clinical trials.

Another thing that was discussed in the patient perspective task force, was the concept of rolls of the different participants. This is not just a decision that a physician makes, or another practitioner, it's important to emphasize the concept of what we call shared decision making. The patient and the patient's family members have to play an important role as we treat patients. Some of these therapies that we use are quite complex and it requires a huge commitment by the patient and their family member. And they really need to understand the facts of those therapies, the implications of those therapies, and have to balance whether or not those therapies are right for them.

Another point that was made, something that I as a physician struggle with, is the concept of palliative care. We heard from our patients in the patient perspective that they want to know at palliative care. They want to know those options. For me personally, as a physician, I always feel like the patients come to us for hope, the patients come to us to get better, and it takes a lot for me to kind of say, maybe we're not doing as well as we would like and maybe we need to consider alternative options. But what we heard from the patients is they want to understand those other options sooner rather than later. And so I think that it is important to the medical community and the patients, as well, to be proactive about making sure we discuss all options of patient care, and sometimes that includes palliative care.

Thank you for joining me, I'm Valerie McLaughlin from the University of Michigan, and I'm aware that I'm rare.

]]>fullEpisode 218 - Vallerie McLaughlin, MDVallerie McLaughlin, MD - World Symposium on Pulmonary HypertensionMon, 04 Feb 2019 13:00:00 +0000In this episode, Vallerie McLaughlin, MD gives an overview of 6th World Symposium on Pulmonary Hypertension, which took place in Nice, France, in early 2018. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field.

]]>In this episode, Vallerie McLaughlin, MD gives an overview of 6th World Symposium on Pulmonary Hypertension, which took place in Nice, France, in early 2018. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field.

]]>10:49cleanblood,research,heart,awareness,therapies,asthma,breathless,rare,ph,clinical,trials,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdiseaseIn this episode, Vallerie McLaughlin, MD gives an overview of 6th World Symposium on Pulmonary Hypertension, which took place in Nice, France, in early 2018. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA).
Learn more about pulmonary hypertension at www.phaware.global @umichmedicine @umichCVC #phaware #phawareMD319fullphaware global associationVikas Aggarwal, MBBS, MPH - phaware® interview 217Fri, 01 Feb 2019 13:00:00 +0000In this episode, Dr. Vikas Aggarwal discusses balloon pulmonary angioplasty as a treatment for CTEPH patients. Dr. Aggarwal is a board certified interventional cardiologist and Endovascular Interventional specialist. He directs the interventional vascular medicine program within the division of cardiology at the University of Michigan Hospital in Ann Arbor. Dr. Aggarwal specializes in percutaneous therapies for Deep Vein and Pulmonary Thromboembolic disease including catheter based thrombus removal and Balloon Pulmonary Angioplasty.

Hi, my name is Dr. Vikas Aggarwal. I'm an international cardiologist at the University of Michigan Hospital in Ann Arbor. My area of expertise happens to be in pulmonary vascular interventions and I'm currently leading the balloon pulmonary angioplasty program for CTEPH patients. I work hands in hand with our multidisciplinary CTEPH team, led by Dr. Valerie McLaughlin, Dr. Scott Visovatti and our cardiac surgeon, Dr. Jonathan Haft.

The University of Michigan Hospital happens to be the lead academic medical center in the state of Michigan. Recently, balloon pulmonary angioplasty was introduced as a new treatment option for patients with chronic thromboembolic pulmonary hypertension who are no longer surgical candidates.

CTEPH stands for chronic thromboembolic pulmonary hypertension. It's a disease that involves blood clots in the arteries supplying both our lungs. These blood clots are leftover clots from prior pulmonary embolisms and are now apparent and organized within the wall of these arteries, which leads to narrowing of the branches of the pulmonary arteries in our lung, leading to increased pressure inside the lungs, which in turn leads to extra stress and effects the right side of our heart.

The treatment of choice for patients with this pathology happens to be cardiac surgery, in which a surgeon dissects down into these branches of the pulmonary artery and takes this clot out. However, more than 50% of all patients with this pathology are not able to get the surgery, due to multiple clinical and anatomical reasons.

Recently, pulmonary angioplasty, which is a minimally invasive procedure in which we pass a wire across these narrowed arteries and use small balloons to open those arteries and to relieve those narrowings, has shown a lot of promise. That's where my experience lies in and that's where we want to make a difference in the care of these patients.

Balloon pulmonary angioplasty was, in fact, first reported from the United States in 2001 by Feinstein, et al, from Boston, Massachusetts. However, that experience showed a significantly high rate of complications and bleeding from this procedure.

However, since then, the Japanese have taken the forefront in this field and have now consistently shown over the past decade that this procedure, if done right, can be performed safely and effectively in such cases. Since the Japanese experience has been more widely known and widely published, experience and interest has been increasing in the United States over the past five years.

I was first introduced to this procedure in 2014 when I was working in Philadelphia at Temple University Hospital, and then over the course of the next four years, I was fortunate to gain more experience and expertise and refine my technique.

Subsequently, I also interacted and now became very good friends with my colleagues at Kyushu University in Fukuoka, Japan and was able to go visit them and learn the Japanese way of doing pulmonary angioplasty hand in hand this past summer. More recently, I'm very excited and fortunate to join the University of Michigan's pulmonary hypertension and CTEPH Center of Excellence and I am able to bring this newer technology to our patients at the University of Michigan.

Currently, the standard of care for patients with CTEPH happens to be cardiac surgery or pulmonary endarterectomy. Balloon pulmonary angioplasty is a relatively new procedure and the Japanese have shown that in most patients with CTEPH, balloon pulmonary angioplasty is often as effective as cardiac surgery, albeit it requires on an average, four to five treatment sessions per patient.

However, in the absence of randomized, well designed clinical trial data, pulmonary endarterectomy continues to be the standard of care in the United States, and balloon pulmonary angioplasty currently is only limited to patients who cannot undergo pulmonary endarterectomy due to clinical or patient anatomical factors.

Our first patient in the state of Michigan that received balloon pulmonary angioplasty at our expert center has had one session and they are scheduled to undergo two or three more sessions over the next few weeks. This patient had severe pulmonary hypertension, despite being on pulmonary hypertension medications, and was noted to have chronic thromboembolic disease on pulmonary imaging. However, her disease was so distal in the subsegmental branches, which were not accessible to cardiac surgery. She was evaluated by our expert surgeon as well as our pulmonary hypertension team and was thought to be a better candidate for pulmonary angioplasty and not pulmonary endarterectomy.

She underwent her first session of the balloon pulmonary angioplasty at our center last month and she's already feeling a lot better compared to how she was feeling prior to the procedure. We plan on performing a few more sessions on her with the goal of normalizing her pulmonary pressures and hopefully getting her off the pulmonary hypertension medications.

When we saw the patient after her first treatment, she was very excited and pleased to see her symptoms get better. She told us that she was able to do more, [was] less short of breath, and that was heartening and exciting to hear.

Our goal is to spread the word about our multidisciplinary expert CTEPH center now here in the state of Michigan, and we hope to take care of patients from all over the Midwest, not just the state of Michigan, as well as nationally, and from Canada, too.

If you have unexplained shortness of breath and limitation in your activity level, you should be seen by either a cardiologist or a pulmonologist, and the workup for this pathology starts with an echocardiogram. If the echocardiogram shows evidence of right heart strain and/or pulmonary hypertension, you should definitely receive a ventilation profusion scan or a V/Q scan to look for chronic thromboembolic pulmonary hypertension as the underlying pathology behind the diagnosis of pulmonary hypertension.

If you happen to have evidence of ventilation profusion mismatch or chronic thromboembolic pulmonary hypertension, you should be referred to an expert center like ours as soon as possible for further evolution and the workup of this disease, which includes right heart catheterization as well as a pulmonary angiogram and a CT angiogram to better define the hemodynamics as well as the anatomy and the obstructive component from this pathology.

The only piece of advice I would give everybody is chronic thromboembolic pulmonary hypertension is common and is often misdiagnosed or is diagnosed very late in the disease pathology, so please, everyone think about chronic thromboembolic pulmonary hypertension in your patients with unexplained dyspnea or dyspnea out of proportion to their medical condition, and please screen your patients for this disease with a V/Q scan.

If you have any questions, we can help you take care of these patients. Please feel free to reach out.

]]>In this episode, Dr. Vikas Aggarwal discusses balloon pulmonary angioplasty as a treatment for CTEPH patients. Dr. Aggarwal is a board certified interventional cardiologist and Endovascular Interventional specialist. He directs the interventional vascular medicine program within the division of cardiology at the University of Michigan Hospital in Ann Arbor. Dr. Aggarwal specializes in percutaneous therapies for Deep Vein and Pulmonary Thromboembolic disease including catheter based thrombus removal and Balloon Pulmonary Angioplasty.

Hi, my name is Dr. Vikas Aggarwal. I'm an international cardiologist at the University of Michigan Hospital in Ann Arbor. My area of expertise happens to be in pulmonary vascular interventions and I'm currently leading the balloon pulmonary angioplasty program for CTEPH patients. I work hands in hand with our multidisciplinary CTEPH team, led by Dr. Valerie McLaughlin, Dr. Scott Visovatti and our cardiac surgeon, Dr. Jonathan Haft.

The University of Michigan Hospital happens to be the lead academic medical center in the state of Michigan. Recently, balloon pulmonary angioplasty was introduced as a new treatment option for patients with chronic thromboembolic pulmonary hypertension who are no longer surgical candidates.

CTEPH stands for chronic thromboembolic pulmonary hypertension. It's a disease that involves blood clots in the arteries supplying both our lungs. These blood clots are leftover clots from prior pulmonary embolisms and are now apparent and organized within the wall of these arteries, which leads to narrowing of the branches of the pulmonary arteries in our lung, leading to increased pressure inside the lungs, which in turn leads to extra stress and effects the right side of our heart.

The treatment of choice for patients with this pathology happens to be cardiac surgery, in which a surgeon dissects down into these branches of the pulmonary artery and takes this clot out. However, more than 50% of all patients with this pathology are not able to get the surgery, due to multiple clinical and anatomical reasons.

Recently, pulmonary angioplasty, which is a minimally invasive procedure in which we pass a wire across these narrowed arteries and use small balloons to open those arteries and to relieve those narrowings, has shown a lot of promise. That's where my experience lies in and that's where we want to make a difference in the care of these patients.

Balloon pulmonary angioplasty was, in fact, first reported from the United States in 2001 by Feinstein, et al, from Boston, Massachusetts. However, that experience showed a significantly high rate of complications and bleeding from this procedure.

However, since then, the Japanese have taken the forefront in this field and have now consistently shown over the past decade that this procedure, if done right, can be performed safely and effectively in such cases. Since the Japanese experience has been more widely known and widely published, experience and interest has been increasing in the United States over the past five years.

I was first introduced to this procedure in 2014 when I was working in Philadelphia at Temple University Hospital, and then over the course of the next four years, I was fortunate to gain more experience and expertise and refine my technique.

Subsequently, I also interacted and now became very good friends with my colleagues at Kyushu University in Fukuoka, Japan and was able to go visit them and learn the Japanese way of doing pulmonary angioplasty hand in hand this past summer. More recently, I'm very excited and fortunate to join the University of Michigan's pulmonary hypertension and CTEPH Center of Excellence and I am able to bring this newer technology to our patients at the University of Michigan.

Currently, the standard of care for patients with CTEPH happens to be cardiac surgery or pulmonary endarterectomy. Balloon pulmonary angioplasty is a relatively new procedure and the Japanese have shown that in most patients with CTEPH, balloon pulmonary angioplasty is often as effective as cardiac surgery, albeit it requires on an average, four to five treatment sessions per patient.

However, in the absence of randomized, well designed clinical trial data, pulmonary endarterectomy continues to be the standard of care in the United States, and balloon pulmonary angioplasty currently is only limited to patients who cannot undergo pulmonary endarterectomy due to clinical or patient anatomical factors.

Our first patient in the state of Michigan that received balloon pulmonary angioplasty at our expert center has had one session and they are scheduled to undergo two or three more sessions over the next few weeks. This patient had severe pulmonary hypertension, despite being on pulmonary hypertension medications, and was noted to have chronic thromboembolic disease on pulmonary imaging. However, her disease was so distal in the subsegmental branches, which were not accessible to cardiac surgery. She was evaluated by our expert surgeon as well as our pulmonary hypertension team and was thought to be a better candidate for pulmonary angioplasty and not pulmonary endarterectomy.

She underwent her first session of the balloon pulmonary angioplasty at our center last month and she's already feeling a lot better compared to how she was feeling prior to the procedure. We plan on performing a few more sessions on her with the goal of normalizing her pulmonary pressures and hopefully getting her off the pulmonary hypertension medications.

When we saw the patient after her first treatment, she was very excited and pleased to see her symptoms get better. She told us that she was able to do more, [was] less short of breath, and that was heartening and exciting to hear.

Our goal is to spread the word about our multidisciplinary expert CTEPH center now here in the state of Michigan, and we hope to take care of patients from all over the Midwest, not just the state of Michigan, as well as nationally, and from Canada, too.

If you have unexplained shortness of breath and limitation in your activity level, you should be seen by either a cardiologist or a pulmonologist, and the workup for this pathology starts with an echocardiogram. If the echocardiogram shows evidence of right heart strain and/or pulmonary hypertension, you should definitely receive a ventilation profusion scan or a V/Q scan to look for chronic thromboembolic pulmonary hypertension as the underlying pathology behind the diagnosis of pulmonary hypertension.

If you happen to have evidence of ventilation profusion mismatch or chronic thromboembolic pulmonary hypertension, you should be referred to an expert center like ours as soon as possible for further evolution and the workup of this disease, which includes right heart catheterization as well as a pulmonary angiogram and a CT angiogram to better define the hemodynamics as well as the anatomy and the obstructive component from this pathology.

The only piece of advice I would give everybody is chronic thromboembolic pulmonary hypertension is common and is often misdiagnosed or is diagnosed very late in the disease pathology, so please, everyone think about chronic thromboembolic pulmonary hypertension in your patients with unexplained dyspnea or dyspnea out of proportion to their medical condition, and please screen your patients for this disease with a V/Q scan.

If you have any questions, we can help you take care of these patients. Please feel free to reach out.

]]>In this episode, Dr. Vikas Aggarwal discusses balloon pulmonary angioplasty as a treatment for CTEPH patients. Dr. Aggarwal is a board certified interventional cardiologist and Endovascular Interventional specialist. He directs the interventional vascular medicine program within the division of cardiology at the University of Michigan Hospital in Ann Arbor. Dr. Aggarwal specializes in percutaneous therapies for Deep Vein and Pulmonary Thromboembolic disease including catheter based thrombus removal and Balloon Pulmonary Angioplasty.

Jonathan Haft, MD discusses PTE (pulmonary thromboendarterectomy) surgery, which is the potentially curative operation offered for some patients with chronic thromboembolic pulmonary hypertension (CTEPH). Dr. Haft has been the director of Michigan’s internationally recognized ECMO program since 2005. He is also the Associate Director of the Cardiovascular Intensive Care unit, and is the Chief of Cardiothoracic Surgery at the Ann Arbor VA Hospital.

My name is Jonathan Haft. I am a physician and cardiothoracic surgeon. I practice at the University of Michigan in Ann Arbor. I've been in practice since 2005.

What I'd like to talk about today is pulmonary thromboendarterectomy, which is the potentially curative operation offered for some patients with chronic thromboembolic pulmonary hypertension. I'd like to talk to you a little bit about the indications for surgery, the operation itself, and then the typical recovery period.

Patients that have chronic thromboembolic pulmonary hypertension have symptoms of breathlessness, exertional intolerance, and often times this leads to an echocardiogram. An echocardiogram or an ultrasound of the heart will identify that the patient has pulmonary hypertension.

The next step is typically a V/Q scan or a ventilation–perfusion scan, which will identify that there are areas of the lung that are under perfused, that there is relative obstruction to blood flow within the lungs. Once we have established that the patient has pulmonary hypertension and that the patient has an abnormal V/Q scan, the next step in testing would be a pulmonary angiogram; where a catheter is inserted through a vein, often a vein in the neck or a vein from the lower extremities, and advanced into the arteries of the lungs, the pulmonary arteries and dye or contrast will be injected to create a roadmap of the blood vessels into the lungs. This test, number one, will tell you if the patient has evidence of CTEPH and most importantly, will identify if the patient is potentially a candidate for surgical removal of these obstructing clots.

When I evaluate a patient in my office, there's a number of factors about the patient that we consider whether or not they could benefit from a pulmonary thromboendarterectomy [PTE]. The first is that the patient actually has physical limitations from the disease. The operation itself is invasive and carries risks of complications, and we want to be certain that the operation is being performed with the intent of improving the quality of their life. So, we want to be sure that the patients actually does have some physical limitations; they've noticed that their physical capacity has declined, and therefore the patients quality of life has declined, and they're interested in pursuing with an aggressive strategy to improve their life. We want to be sure that there is substantial burden of obstructing clot that would be surgically accessible.

If we think about the circulation to the lungs, there is the main pulmonary artery, that's the artery that exits the heart that is delivering blood to both lungs. Then there are the branch pulmonary arteries, one for the right, and one for the left. Then there are the lobar branches heading to the upper lobe, the middle lobe, and the lower lobe. There are the segmental branches, heading to the 10 segments that are present in each lung. Then there are a multitude of subsegmental branches beyond the segments.

For the most part, when patients have substantial obstruction that originates at the level of the segments, the segmental level, an experienced surgeon should be able to access that obstructing material and therefore the patient would be a reasonable candidate to proceed with pulmonary endarterectomy. But again, it really depends upon the imaging, and being able to identify the extent and location of the obstructing thrombus, as well as the experience of the surgeon.

The last factor when we evaluate a patient is the patient themselves. Is the patient going to be physically able to withstand an extensive open heart operation? If the patient is too old, or has too many other medical problems, then they may be more at risk of suffering complication.

Let me walk you through the operation, how it's performed, and then we can talk about the postoperative recovery.

The operation is open heart surgery, so again we expose the heart and the blood vessels of the lungs through an incision in the breast bone, we call it a median sternotomy. It's a vertical incision through the breast bone. Obviously, the patient is under full anesthesia and is unaware what's happening during the operation. Then what we do is place the patient on the heart-lung bypass machine, or cardiopulmonary bypass, by placing catheters into the superior and inferior vena cava. Those are the big veins that drain all the blood from the upper and the lower half of the body. We also place a catheter into the aorta, and that's to infuse the blood. Therefore, bypassing all the blood that would normally travel through the heart.

Now the patient is on the heart-lung bypass machine, we can then deliver what we call cardioplegia. It's a cold preservative solution that goes directly into the heart and it induces cardiac quiescence, so the heart is not moving. Under those circumstances, we can now open up the arteries that are traveling to the right and to the left lung, to see where the obstructing clot might be.

Now [we see] if the clot has undergone a process of organization, where the clot has now turned into scar. By turning into scar, it has now grown into the wall of the blood vessel. It is now incorporated within the blood vessel, so when I open up these arteries it's not just a matter of reaching in there and trying to pluck out that obstructing clot. That's something that might work for a new pulmonary embolism, where it's just clot that's sitting in the obstructing blood vessel. This is clot that has again, has organized and has grown into the wall. The only way for the clot to come out, would be to peel it out. What I explain to patients, it's akin to peeling the rind off an orange. Where you have to get into that right space, and as you peel that rind off, you're always looking to make sure that you're in that right space. So we identify where the obstructing clot is on the inside of the artery, and now we have to very carefully peel it from the wall on the inside of the artery. We have to follow that into all the different branches within the pulmonary artery. Now it is a tedious procedure, and requires a fair amount of meticulous attention to detail. Most importantly, you have to very carefully be able to see, precisely see into all of these branches.

When you're working on the inside of a blood vessel, there tends to be blood on the inside of a blood vessel, and therefore that blood obscures your ability to precisely see those segmental and subsegmental branches. We have to eliminate this blood for better visualization. The way we do that is with a technique called hypothermic circulatory arrest. What this means is that we use the heart-lung machine, or the cardiopulmonary bypass system, to very aggressively cool the blood. By cooling the blood, the entire body, the brain, all the other vital organs, become very, very cold and by making them cold, that reduces the body's metabolism, the metabolic rate. Therefore, the body is consuming very little nutrients and very little oxygen. That allows us, for brief periods of time, to completely stop all circulation. We halt the circulation throughout the body, for brief periods of time, so that we can now visualize on the inside of those blood vessels where the obstructing clot is extending. We then perform our dissection.

We typically limit the period of no circulation to no more than 20 minutes. At 20 minutes we reestablish circulation, reperfuse, or once again deliver blood throughout the body. Once we have completed all of our dissection, then we begin the process of rewarming the patient. Once the patient is warm, and the metabolic rate starts to rise, the heart starts to beat again and then we're able to separate the patient from the heart-lung machine.

Once we finish the operation, and we close the patient up, they are transported directly to an intensive care unit. At our center, specifically unique for this operation, we tend to leave the patient tranquilized and on a ventilator overnight and begin the process the following day to liberate them from the ventilator. That's a process of withholding the sedation, so the patient is now awake and interactive. Once we feel the patient is able to breath on their own, the breathing tube is removed and the patient is now breathing on their own. At our center, we typically do that the following morning after surgery.

It is very important for these patients to fully expand their lungs. These patients have been exposed to obstructive blood flow throughout the lungs, and therefore there's going to be new robust blood flow throughout the lungs. That can cause a phenomenon that we refer to as reperfusion edema. Edema means swelling. So now, you've removed the obstruction and the lung is going to get a tremendous amount of blood flow that it has not seen oftentimes for many years, and the lung may swell because it's unprepared to have all this circulation. So it's very important that we get these patients to take as deep a breath as possible, so we can fill their lungs with as much air, and therefore expanding their lungs and eliminating some of this edema, or extra fluid in the lungs. This is, of course, challenging for patients after surgery, because they have pain from the incision. But mostly we instruct, we educate that is going to be important that they really work on expanding their lungs.

We also initiate these patients on anticoagulation, as soon as we feel it's safe. Though all of these patients were taking some form of anticoagulation before surgery. That anticoagulation is withheld, typically the night before the operation. But the following day, once we're confident there's no evidence of residual bleeding from surgery, we initiate anticoagulation, typically starting with heparin, and then transitioning to an oral agent, most commonly warfarin or Coumadin. At our center, we do tell patients that removing these obstructing clots do not eliminate their needs for anticoagulation. These patients have demonstrated that they have the likelihood to make blood clots, and therefore we recommend that they remain on anticoagulation for the rest of their life.

For this operation, the average length of stay in the hospital is typically around 10 days. Most patients, it takes them about a month until they really feel like they have the wind in their sails again. The vast majority of patients at one to two months have substantial improvement in their baseline symptoms, which continues to improve with postoperative rehabilitation.

Chronic thromboembolic pulmonary hypertension is a mechanical problem, and therefore this condition is potentially curable with an operation. Many of these patients who have a successful operation, are effectively cured of the disease and the symptomatic improvement that they experience is transformative. For me, it is very rewarding to take patients that are suffering from this disease, and then be able to restore really what is normalcy to their life.

Jonathan Haft, MD discusses PTE (pulmonary thromboendarterectomy) surgery, which is the potentially curative operation offered for some patients with chronic thromboembolic pulmonary hypertension (CTEPH). Dr. Haft has been the director of Michigan’s internationally recognized ECMO program since 2005. He is also the Associate Director of the Cardiovascular Intensive Care unit, and is the Chief of Cardiothoracic Surgery at the Ann Arbor VA Hospital. My name is Jonathan Haft. I am a physician and cardiothoracic surgeon. I practice at the University of Michigan in Ann Arbor. I've been in practice since 2005.

What I'd like to talk about today is pulmonary thromboendarterectomy, which is the potentially curative operation offered for some patients with chronic thromboembolic pulmonary hypertension. I'd like to talk to you a little bit about the indications for surgery, the operation itself, and then the typical recovery period.

Patients that have chronic thromboembolic pulmonary hypertension have symptoms of breathlessness, exertional intolerance, and often times this leads to an echocardiogram. An echocardiogram or an ultrasound of the heart will identify that the patient has pulmonary hypertension.

The next step is typically a V/Q scan or a ventilation–perfusion scan, which will identify that there are areas of the lung that are under perfused, that there is relative obstruction to blood flow within the lungs. Once we have established that the patient has pulmonary hypertension and that the patient has an abnormal V/Q scan, the next step in testing would be a pulmonary angiogram; where a catheter is inserted through a vein, often a vein in the neck or a vein from the lower extremities, and advanced into the arteries of the lungs, the pulmonary arteries and dye or contrast will be injected to create a roadmap of the blood vessels into the lungs. This test, number one, will tell you if the patient has evidence of CTEPH and most importantly, will identify if the patient is potentially a candidate for surgical removal of these obstructing clots.

When I evaluate a patient in my office, there's a number of factors about the patient that we consider whether or not they could benefit from a pulmonary thromboendarterectomy [PTE]. The first is that the patient actually has physical limitations from the disease. The operation itself is invasive and carries risks of complications, and we want to be certain that the operation is being performed with the intent of improving the quality of their life. So, we want to be sure that the patients actually does have some physical limitations; they've noticed that their physical capacity has declined, and therefore the patients quality of life has declined, and they're interested in pursuing with an aggressive strategy to improve their life. We want to be sure that there is substantial burden of obstructing clot that would be surgically accessible.

If we think about the circulation to the lungs, there is the main pulmonary artery, that's the artery that exits the heart that is delivering blood to both lungs. Then there are the branch pulmonary arteries, one for the right, and one for the left. Then there are the lobar branches heading to the upper lobe, the middle lobe, and the lower lobe. There are the segmental branches, heading to the 10 segments that are present in each lung. Then there are a multitude of subsegmental branches beyond the segments.

For the most part, when patients have substantial obstruction that originates at the level of the segments, the segmental level, an experienced surgeon should be able to access that obstructing material and therefore the patient would be a reasonable candidate to proceed with pulmonary endarterectomy. But again, it really depends upon the imaging, and being able to identify the extent and location of the obstructing thrombus, as well as the experience of the surgeon.

The last factor when we evaluate a patient is the patient themselves. Is the patient going to be physically able to withstand an extensive open heart operation? If the patient is too old, or has too many other medical problems, then they may be more at risk of suffering complication.

Let me walk you through the operation, how it's performed, and then we can talk about the postoperative recovery.

The operation is open heart surgery, so again we expose the heart and the blood vessels of the lungs through an incision in the breast bone, we call it a median sternotomy. It's a vertical incision through the breast bone. Obviously, the patient is under full anesthesia and is unaware what's happening during the operation. Then what we do is place the patient on the heart-lung bypass machine, or cardiopulmonary bypass, by placing catheters into the superior and inferior vena cava. Those are the big veins that drain all the blood from the upper and the lower half of the body. We also place a catheter into the aorta, and that's to infuse the blood. Therefore, bypassing all the blood that would normally travel through the heart.

Now the patient is on the heart-lung bypass machine, we can then deliver what we call cardioplegia. It's a cold preservative solution that goes directly into the heart and it induces cardiac quiescence, so the heart is not moving. Under those circumstances, we can now open up the arteries that are traveling to the right and to the left lung, to see where the obstructing clot might be.

Now [we see] if the clot has undergone a process of organization, where the clot has now turned into scar. By turning into scar, it has now grown into the wall of the blood vessel. It is now incorporated within the blood vessel, so when I open up these arteries it's not just a matter of reaching in there and trying to pluck out that obstructing clot. That's something that might work for a new pulmonary embolism, where it's just clot that's sitting in the obstructing blood vessel. This is clot that has again, has organized and has grown into the wall. The only way for the clot to come out, would be to peel it out. What I explain to patients, it's akin to peeling the rind off an orange. Where you have to get into that right space, and as you peel that rind off, you're always looking to make sure that you're in that right space. So we identify where the obstructing clot is on the inside of the artery, and now we have to very carefully peel it from the wall on the inside of the artery. We have to follow that into all the different branches within the pulmonary artery. Now it is a tedious procedure, and requires a fair amount of meticulous attention to detail. Most importantly, you have to very carefully be able to see, precisely see into all of these branches.

When you're working on the inside of a blood vessel, there tends to be blood on the inside of a blood vessel, and therefore that blood obscures your ability to precisely see those segmental and subsegmental branches. We have to eliminate this blood for better visualization. The way we do that is with a technique called hypothermic circulatory arrest. What this means is that we use the heart-lung machine, or the cardiopulmonary bypass system, to very aggressively cool the blood. By cooling the blood, the entire body, the brain, all the other vital organs, become very, very cold and by making them cold, that reduces the body's metabolism, the metabolic rate. Therefore, the body is consuming very little nutrients and very little oxygen. That allows us, for brief periods of time, to completely stop all circulation. We halt the circulation throughout the body, for brief periods of time, so that we can now visualize on the inside of those blood vessels where the obstructing clot is extending. We then perform our dissection.

We typically limit the period of no circulation to no more than 20 minutes. At 20 minutes we reestablish circulation, reperfuse, or once again deliver blood throughout the body. Once we have completed all of our dissection, then we begin the process of rewarming the patient. Once the patient is warm, and the metabolic rate starts to rise, the heart starts to beat again and then we're able to separate the patient from the heart-lung machine.

Once we finish the operation, and we close the patient up, they are transported directly to an intensive care unit. At our center, specifically unique for this operation, we tend to leave the patient tranquilized and on a ventilator overnight and begin the process the following day to liberate them from the ventilator. That's a process of withholding the sedation, so the patient is now awake and interactive. Once we feel the patient is able to breath on their own, the breathing tube is removed and the patient is now breathing on their own. At our center, we typically do that the following morning after surgery.

It is very important for these patients to fully expand their lungs. These patients have been exposed to obstructive blood flow throughout the lungs, and therefore there's going to be new robust blood flow throughout the lungs. That can cause a phenomenon that we refer to as reperfusion edema. Edema means swelling. So now, you've removed the obstruction and the lung is going to get a tremendous amount of blood flow that it has not seen oftentimes for many years, and the lung may swell because it's unprepared to have all this circulation. So it's very important that we get these patients to take as deep a breath as possible, so we can fill their lungs with as much air, and therefore expanding their lungs and eliminating some of this edema, or extra fluid in the lungs. This is, of course, challenging for patients after surgery, because they have pain from the incision. But mostly we instruct, we educate that is going to be important that they really work on expanding their lungs.

We also initiate these patients on anticoagulation, as soon as we feel it's safe. Though all of these patients were taking some form of anticoagulation before surgery. That anticoagulation is withheld, typically the night before the operation. But the following day, once we're confident there's no evidence of residual bleeding from surgery, we initiate anticoagulation, typically starting with heparin, and then transitioning to an oral agent, most commonly warfarin or Coumadin. At our center, we do tell patients that removing these obstructing clots do not eliminate their needs for anticoagulation. These patients have demonstrated that they have the likelihood to make blood clots, and therefore we recommend that they remain on anticoagulation for the rest of their life.

For this operation, the average length of stay in the hospital is typically around 10 days. Most patients, it takes them about a month until they really feel like they have the wind in their sails again. The vast majority of patients at one to two months have substantial improvement in their baseline symptoms, which continues to improve with postoperative rehabilitation.

Chronic thromboembolic pulmonary hypertension is a mechanical problem, and therefore this condition is potentially curable with an operation. Many of these patients who have a successful operation, are effectively cured of the disease and the symptomatic improvement that they experience is transformative. For me, it is very rewarding to take patients that are suffering from this disease, and then be able to restore really what is normalcy to their life.

]]>In this episode, Jonathan Haft, MD discusses PTE (pulmonary thromboendarterectomy) surgery, which is the potentially curative operation offered for some patients with chronic thromboembolic pulmonary hypertension (CTEPH). Dr. Haft has been the director of Michigan’s internationally recognized ECMO program since 2005. He is also the Associate Director of the Cardiovascular Intensive Care unit, and is the Chief of Cardiothoracic Surgery at the Ann Arbor VA Hospital.

]]>10:25cleanblood,early,research,heart,awareness,asthma,iv,breathless,rare,ph,diagnosis,pulmonary,raredisease,catheterization,clinicaltrials,pulmonaryhypertension,phaware,cteph,lungdisease,thromboendarterectomyJonathan Haft, MD has been the director of Michigan’s internationally recognized ECMO program since 2005. He is also the Associate Director of the Cardiovascular Intensive Care unit, and is the Chief of Cardiothoracic Surgery at the Ann Arbor VA Hospital. Dr. Haft discusses PTE (pulmonary thromboendarterectomy) surgery, which is the potentially curative operation offered for some patients with chronic thromboembolic pulmonary hypertension (CTEPH). www.phaware.global @umichmedicine #phaware #phawareMD
317fullphaware global associationMarc de Perrot, MD phaware® interview 215Fri, 25 Jan 2019 13:00:00 +0000

Dr. Marc de Perrot joined the Division of Thoracic Surgery as a staff surgeon and Assistant Professor at the University of Toronto in 2005. In 2009, he was appointed Associate Professor of Surgery based on his research and clinical work. Dr. de Perrot’s surgical expertise focuses on surgery for end-stage lung diseases such as lung and heart-lung transplantation as well as pulmonary endarterectomy for chronic thromboembolic pulmonary hypertension. He is also the Head of the Toronto Mesothelioma Program and is involved in basic science research, new treatment with immunotherapy and combined modality therapy. In this episode, Dr. de Perrot discusses the importance of early diagnosis.

I'm Marc de Perrot, I'm a thoracic surgeon at the Toronto General Hospital. I'm in charge of the chronic thromboembolic pulmonary hypertension activity, at the Toronto General Hospital, University Health Network. I'm a thoracic surgeon performing pulmonary thromboendarterectomy as a treatment for chronic thromboembolic pulmonary hypertension, or CTEPH.

I've been working in the field of pulmonary hypertension for almost 20 years. I initially started working in the context of pulmonary hypertension as a lung transplant surgeon; that's how I got involved with PH. Then we were seeing some patients with chronic thromboembolic pulmonary hypertension [who were] referred for lung transplantation. The surgery of pulmonary thromboendarterectomy has improved in success. Eventually, it was becoming the treatment of choice. That's how I got involved more directly in CTEPH.

The pulmonary endarterectomy is a curative treatment CTEPH. It was a much better option than the lung transplantation, which has some difficulty and some limitation in the long term. So the pulmonary endarterectomy, or thromboendarterectomy, was clearly a much better option for patients with CTEPH. It was important that the patients were referred for the right surgery. That's how I started the pulmonary thromboendarterectomy program at the Toronto General Hospital, back in 2005.

The meaning of chronic thromboembolic pulmonary hypertension, or CTEPH is two things: one is the pulmonary hypertension, and the cause of pulmonary hypertension is chronic thromboembolic disease. So these are patients who have a history of pulmonary emboli. The emboli leads to scarring of the vessel of the lung, which eventually leads to the pulmonary hypertension. So it's really the combination of the pulmonary emboli and the development of pulmonary hypertension that leads to the diagnosis.

The important part is that chronic thromboembolic pulmonary hypertension, or CTEPH, is the only curative form of pulmonary hypertension. Therefore it is critical that it is diagnosed properly in order to give a chance of cure to patients with PH, which they might not have otherwise.

CTEPH, like any form of pulmonary hypertension, the symptoms are not very specific. So the main symptoms is shortness of breath, or dyspnea. Patients feel short of breath going up a flight of stairs. A lot of the time, they will attribute that to some deconditioning, or getting older. So to make the diagnosis of CTEPH sometimes takes a long time. It can take about a year for [the eventual] diagnosis to be made. That is similar to pulmonary hypertension in the sense the symptoms are very non-specific and very similar. The difference in CTEPH compared to other patients with PH is that CTEPH patients typically are in their 50s, 60s or 70 years old, or sometimes 80 years old [when they] develop the disease.

So it's a condition that tends to be developing later on in life, in contrast to pulmonary arterial hypertension, which can occur more frequently in younger patients. So, a lot of the time the symptoms are quite similar to patients with emphysema, COPD, pulmonary fibrosis, so the differential diagnosis of CTEPH doesn't necessarily come up at the top of the list. It does take some time for the patient to be diagnosed, occasionally.

On the other hand, once anybody who has a diagnosis of pulmonary hypertension needs to be evaluated for CTEPH, in order to ensure that the diagnosis of CTEPH is made if pulmonary hypertension is diagnosed. Again, since this is the only form of PH that is potentially curative.

The symptoms of shortness of breath is very non-specific, and a lot of the time shortness of breath leads to evaluation of the lungs. So the lung is looked at very carefully, but the vessel of the lungs takes extra investigation in order to be evaluated. So unless we evaluate the vasculature of the lungs, you may miss the diagnosis of pulmonary hypertension, despite the fact that the symptoms are shortness of breath. So anybody with shortness of breath should be evaluated for their lung function, but also from the pulmonary vascular perspective. For the CTEPH part, the main initial test is a ventilation-perfusion scan. So you assess the perfusion of the lung, and you assess the ventilation of the lung, and if you see a mismatch of the perfusion, then you start the investigation for the potential diagnosis of CTEPH.

It's not something that physicians think of as a potential cause of shortness of breath. So one of the critical part is to increase awareness about the possibility of CTEPH as being a cause of dyspnea. The second step is to do the right test to rule in or rule out CTEPH, and the first step is to do a ventilation-perfusion scan, which is really the best test to have a screening tool to evaluate the possibility of CTEPH. So it's really to think about the diagnosis and then do the right test in order to make your diagnosis. If the ventilation-perfusion scan is abnormal that is enough to warrant a referral to a specialist center for treating patients with CTEPH.

The early diagnosis of CTEPH, or pulmonary hypertension is quite critical, in the sense that the evolution of the disease has an impact on the heart. Patients eventually develop heart failure as part of the natural progression of the disease. The early diagnosis of pulmonary hypertension allows the treatment to be started before the heart failure starts to become a problem, and the success of the treatment is much better. From a CTEPH perspective, this is even more critical in the sense that the CTEPH is treated with surgery, the pulmonary thromboendarterectomy, and development of the heart failure increases the risk of the surgery by up to 10 fold.

Early diagnosis of CTEPH can lead to much better results from a curative perspective, with the pulmonary endarterectomy. It also limits the risk of the surgery, so the surgery is much safer, and the success in the long term is much better from a curative perspective.

The diagnosis of CTEPH is potentially linked to pulmonary emboli. There's a lot of research to be done in order to follow some patients with pulmonary emboli, an acute event, in order to make sure that the resolution of the pulmonary emboli is complete. And if not, to be able to make an earlier diagnosis of CTEPH and potentially prevent the development of the pulmonary hypertension once you've had the blood clot.

CTEPH currently is treated permanently with the surgery, the pulmonary thromboendarterectomy. We, in the field of CTEPH, are developing new technique and technology in order to treat the disease without the surgery with minimally invasive approach. So we can go into the vessel of the lung, inflate a balloon in order to enlarge the vessel, and to potentially also cure patients with CTEPH with a minimally invasive approach. So this is a new field that is developing There is a lot of potential development in that direction in order to potentially cure the disease without the surgery, but with a minimally invasive treatment.

As the disease is diagnosed earlier, the success of these new technologies certainly is going to become much better as well in order to prevent the development of pulmonary hypertension. Potentially even, if the disease is caught at a stage where the pulmonary hypertension is not yet even present, but we can see the parameters that potentially will lead to pulmonary hypertension. So that's what the field is progressively evolving towards.

If you have somebody with shortness of breath, you should think of pulmonary vascular disease, and investigate it with either a CT pulmonary angiogram, or a V/Q scan as part of your evaluation on echocardiogram, as well as part of the evaluation for pulmonary hypertension. So anybody with shortness of breath, unless there’s a very clear cause for the shortness of breath, should have an echocardiogram on the evaluation of the pulmonary vascular disease, with some imaging.

Dr. Marc de Perrot joined the Division of Thoracic Surgery as a staff surgeon and Assistant Professor at the University of Toronto in 2005. In 2009, he was appointed Associate Professor of Surgery based on his research and clinical work. Dr. de Perrot’s surgical expertise focuses on surgery for end-stage lung diseases such as lung and heart-lung transplantation as well as pulmonary endarterectomy for chronic thromboembolic pulmonary hypertension. He is also the Head of the Toronto Mesothelioma Program and is involved in basic science research, new treatment with immunotherapy and combined modality therapy. In this episode, Dr. de Perrot discusses the importance of early diagnosis.

I'm Marc de Perrot, I'm a thoracic surgeon at the Toronto General Hospital. I'm in charge of the chronic thromboembolic pulmonary hypertension activity, at the Toronto General Hospital, University Health Network. I'm a thoracic surgeon performing pulmonary thromboendarterectomy as a treatment for chronic thromboembolic pulmonary hypertension, or CTEPH.

I've been working in the field of pulmonary hypertension for almost 20 years. I initially started working in the context of pulmonary hypertension as a lung transplant surgeon; that's how I got involved with PH. Then we were seeing some patients with chronic thromboembolic pulmonary hypertension [who were] referred for lung transplantation. The surgery of pulmonary thromboendarterectomy has improved in success. Eventually, it was becoming the treatment of choice. That's how I got involved more directly in CTEPH.

The pulmonary endarterectomy is a curative treatment CTEPH. It was a much better option than the lung transplantation, which has some difficulty and some limitation in the long term. So the pulmonary endarterectomy, or thromboendarterectomy, was clearly a much better option for patients with CTEPH. It was important that the patients were referred for the right surgery. That's how I started the pulmonary thromboendarterectomy program at the Toronto General Hospital, back in 2005.

The meaning of chronic thromboembolic pulmonary hypertension, or CTEPH is two things: one is the pulmonary hypertension, and the cause of pulmonary hypertension is chronic thromboembolic disease. So these are patients who have a history of pulmonary emboli. The emboli leads to scarring of the vessel of the lung, which eventually leads to the pulmonary hypertension. So it's really the combination of the pulmonary emboli and the development of pulmonary hypertension that leads to the diagnosis.

The important part is that chronic thromboembolic pulmonary hypertension, or CTEPH, is the only curative form of pulmonary hypertension. Therefore it is critical that it is diagnosed properly in order to give a chance of cure to patients with PH, which they might not have otherwise.

CTEPH, like any form of pulmonary hypertension, the symptoms are not very specific. So the main symptoms is shortness of breath, or dyspnea. Patients feel short of breath going up a flight of stairs. A lot of the time, they will attribute that to some deconditioning, or getting older. So to make the diagnosis of CTEPH sometimes takes a long time. It can take about a year for [the eventual] diagnosis to be made. That is similar to pulmonary hypertension in the sense the symptoms are very non-specific and very similar. The difference in CTEPH compared to other patients with PH is that CTEPH patients typically are in their 50s, 60s or 70 years old, or sometimes 80 years old [when they] develop the disease.

So it's a condition that tends to be developing later on in life, in contrast to pulmonary arterial hypertension, which can occur more frequently in younger patients. So, a lot of the time the symptoms are quite similar to patients with emphysema, COPD, pulmonary fibrosis, so the differential diagnosis of CTEPH doesn't necessarily come up at the top of the list. It does take some time for the patient to be diagnosed, occasionally. On the other hand, once anybody who has a diagnosis of pulmonary hypertension needs to be evaluated for CTEPH, in order to ensure that the diagnosis of CTEPH is made if pulmonary hypertension is diagnosed. Again, since this is the only form of PH that is potentially curative.

The symptoms of shortness of breath is very non-specific, and a lot of the time shortness of breath leads to evaluation of the lungs. So the lung is looked at very carefully, but the vessel of the lungs takes extra investigation in order to be evaluated. So unless we evaluate the vasculature of the lungs, you may miss the diagnosis of pulmonary hypertension, despite the fact that the symptoms are shortness of breath. So anybody with shortness of breath should be evaluated for their lung function, but also from the pulmonary vascular perspective. For the CTEPH part, the main initial test is a ventilation-perfusion scan. So you assess the perfusion of the lung, and you assess the ventilation of the lung, and if you see a mismatch of the perfusion, then you start the investigation for the potential diagnosis of CTEPH.

It's not something that physicians think of as a potential cause of shortness of breath. So one of the critical part is to increase awareness about the possibility of CTEPH as being a cause of dyspnea. The second step is to do the right test to rule in or rule out CTEPH, and the first step is to do a ventilation-perfusion scan, which is really the best test to have a screening tool to evaluate the possibility of CTEPH. So it's really to think about the diagnosis and then do the right test in order to make your diagnosis. If the ventilation-perfusion scan is abnormal that is enough to warrant a referral to a specialist center for treating patients with CTEPH.

The early diagnosis of CTEPH, or pulmonary hypertension is quite critical, in the sense that the evolution of the disease has an impact on the heart. Patients eventually develop heart failure as part of the natural progression of the disease. The early diagnosis of pulmonary hypertension allows the treatment to be started before the heart failure starts to become a problem, and the success of the treatment is much better. From a CTEPH perspective, this is even more critical in the sense that the CTEPH is treated with surgery, the pulmonary thromboendarterectomy, and development of the heart failure increases the risk of the surgery by up to 10 fold.

Early diagnosis of CTEPH can lead to much better results from a curative perspective, with the pulmonary endarterectomy. It also limits the risk of the surgery, so the surgery is much safer, and the success in the long term is much better from a curative perspective.

The diagnosis of CTEPH is potentially linked to pulmonary emboli. There's a lot of research to be done in order to follow some patients with pulmonary emboli, an acute event, in order to make sure that the resolution of the pulmonary emboli is complete. And if not, to be able to make an earlier diagnosis of CTEPH and potentially prevent the development of the pulmonary hypertension once you've had the blood clot.

CTEPH currently is treated permanently with the surgery, the pulmonary thromboendarterectomy. We, in the field of CTEPH, are developing new technique and technology in order to treat the disease without the surgery with minimally invasive approach. So we can go into the vessel of the lung, inflate a balloon in order to enlarge the vessel, and to potentially also cure patients with CTEPH with a minimally invasive approach. So this is a new field that is developing There is a lot of potential development in that direction in order to potentially cure the disease without the surgery, but with a minimally invasive treatment.

As the disease is diagnosed earlier, the success of these new technologies certainly is going to become much better as well in order to prevent the development of pulmonary hypertension. Potentially even, if the disease is caught at a stage where the pulmonary hypertension is not yet even present, but we can see the parameters that potentially will lead to pulmonary hypertension. So that's what the field is progressively evolving towards.

If you have somebody with shortness of breath, you should think of pulmonary vascular disease, and investigate it with either a CT pulmonary angiogram, or a V/Q scan as part of your evaluation on echocardiogram, as well as part of the evaluation for pulmonary hypertension. So anybody with shortness of breath, unless there’s a very clear cause for the shortness of breath, should have an echocardiogram on the evaluation of the pulmonary vascular disease, with some imaging.

]]>fullEpisode 215 - Marc de Perrot, MDMarc de Perrot, MD - Early CTEPH DiagnosisThu, 24 Jan 2019 13:00:00 +0000Dr. Marc de Perrot joined the Division of Thoracic Surgery as a staff surgeon and Assistant Professor at the University of Toronto in 2005. In 2009, he was appointed Associate Professor of Surgery based on his research and clinical work. Dr. de Perrot’s surgical expertise focuses on surgery for end-stage lung diseases such as lung and heart-lung transplantation as well as pulmonary endarterectomy for chronic thromboembolic pulmonary hypertension. He is also the Head of the Toronto Mesothelioma Program and is involved in basic science research, new treatment with immunotherapy and combined modality therapy. In this episode, Dr. de Perrot discusses the importance of early diagnosis for CTEPH patients.

]]>Dr. Marc de Perrot joined the Division of Thoracic Surgery as a staff surgeon and Assistant Professor at the University of Toronto in 2005. In 2009, he was appointed Associate Professor of Surgery based on his research and clinical work. Dr. de Perrot’s surgical expertise focuses on surgery for end-stage lung diseases such as lung and heart-lung transplantation as well as pulmonary endarterectomy for chronic thromboembolic pulmonary hypertension. He is also the Head of the Toronto Mesothelioma Program and is involved in basic science research, new treatment with immunotherapy and combined modality therapy. In this episode, Dr. de Perrot discusses the importance of early diagnosis for CTEPH patients.

]]>09:21cleanblood,canada,early,research,heart,awareness,asthma,iv,breathless,rare,ph,diagnosis,pulmonary,raredisease,catheterization,pha,clinicaltrials,pulmonaryhypertension,phaware,lungdiseaseDr. Marc de Perrot joined the Division of Thoracic Surgery as a staff surgeon and Assistant Professor at the University of Toronto in 2005. In 2009, he was appointed Associate Professor of Surgery based on his research and clinical work. In this episodes, Dr. de Perrot discusses the importance of early diagnosis.
Learn more about pulmonary hypertension at www.phaware.global. Learn more about @PHACanada Early Diagnosis Campaign at www.sometimesitsph.ca #phaware #phawareMD316fullphaware global association Angela Bates, MD - phaware® interview 214Tue, 22 Jan 2019 13:00:00 +0000

Dr. Angela Bates is pediatric PH specialist from Stollery Children’s Hospital. She is committed to conducting research in pediatric patients in the areas of critical care, specifically those with congenital heart disease and those affected by infectious diseases in the critical care arena, as well as patients presenting with pulmonary hypertension of all etiologies. Her research is focused on improving diagnosis, treatment, outcomes and resources available to these patients. In this episode, Dr. Bates discusses the importance of early diagnosis.

I'm Dr. Angela Bates. I work at the Stollery Children's Hospital in Edmonton, Alberta, Canada. I take care of the pediatric pulmonary hypertension program there. I'm the medical lead, as well as I do pediatric cardiac ICU. So I see both inpatient and outpatient kiddos, all across Western Canada.

I think pulmonary hypertension in kids - the thing that sets it apart from other lung diseases is we talk about being subclinical, meaning a lot of these kids can live with this disease without having any symptoms. By the time they actually show up, they can be very sick. They can be as sick as being in the ICU. They can show even as sick as dying from this disease. We have a this window of opportunity to make a huge difference with all available drugs that we have now-a-days including oral medications which we didn't have 10 or 15 years ago.

The other thing that's really remarkable is that this is an amazing group of children that really can do a lot despite being quite sick, if you look at their echocardiograms and look at some of their other different investigations. That's kind of remarkable with these kids. Again, [it can] also kind of trick us practitioners in terms of how aggressive we are with treatment. I think sometimes we aren't thinking about screening them, we're not thinking doing an ECG or ECHO. They give us very few clues early on.

Also a lot of our gold standard of tests are invasive. The gold standard would be doing a cardiac catheterization which requires sedation, a general anesthetic. It requires actually putting catheters into the groin. Those are all things that sound scary to parents, scary to kids, and we respect the fact that some of these kiddos; they have a high risk of something going bad in these procedures. So I think it makes it a bit difficult. That being said, we have really come a long way in terms of noninvasive investigations. Our ECHO technology is better. We now have cardiac MRI and everyday people in the radiology world are looking at trying to find more reliable noninvasive tools.

For us the challenge is a few things. For one, our group of pediatric pulmonary hypertension is very diverse. So two big groups are very different. You have these arterial pulmonary hypertension kiddos. They can either be hereditary or idiopathic related heart disease or other things like connective tissue. Then you have another group of developmental lung diseases. Even [with] treatments. Are there certain treatments that are useful in one group versus another? Some of these kiddos are very little, looking at non-invasive techniques of diagnosing and following these kids is very challenging. That's really where a lot of our research is headed now-a-days.

Then just even clinical end points. Because it's hard to really identify what matters the most for these kids. Obviously, yes, survival matters, but is there something that we can follow as a marker that's a little bit more sensitive? That we can follow that will change the earlier on disease progression that will help us to modify the therapy. Then again another thing that's just a little bit challenging is even different drugs that are available. So, oral medication we do have a lot more. A lot of them aren't approved in pediatrics, so we certainly use lots of medications off label but even what's available for them to get at home. Whether it's subcutaneous delivered or IV and so forth. That's some of the challenges, but also exciting areas for us to move forward in PH.

If you look back at the older literature, diagnosing kids late in the game - their prognosis was quite terrible. Median survival without treatment was around 18 months. Now that we know that introducing different therapies earlier on, these kiddos can live 10-15 years, which gives them that window that; one to obviously be kids and do well but, two to get to a point where maybe lung transplant is a better option than when they were 5 years of age. It also gives us more time to find better treatments. Certainly implementing therapies early on, not only improves their survival but just their quality of life which really is what's most important to parents, to kids, and as well as to us as health care professionals.

In terms of advice for a general practitioner or a pediatrician in the community following these kiddos is, even though it's a scary disease educating yourself is very important. Empowering the families, listening to the families. And then, always not being afraid to ask advice. We're never too busy to talk on the phone. We are never too busy to help follow these kids in the community with you, to see them in our clinic, to provide more education.

The biggest thing for these kiddos is quality of life. Although sometimes we may have to sort of negotiate what sort of therapies are best in these kids. Really in the end, these kiddos should be at home with their families, out doing activities that they enjoy. We work within those constraints but to truly focus on how can kids best live out their childhood.

If you have a kiddo that just doesn't fit the normal mold of what you're used to seeing for common childhood illnesses like asthma or that sort of thing, think about pulmonary hypertension. And if your tests don’t make sense, certainly think of pulmonary hypertension. And I think the other thing is you can do a simple cardiac exam. If you can feel a second heart sound that's abnormal, if the kiddo looks like there's a right heart failure, there's a reason for that. A simple thing like an ECG is a great place to start. There is a lot of things you can do in the office to screen these kiddos and then pick up the phone.

The field of pulmonary hypertension both in the pediatric and the adult world is always moving forward and we really try to empower our families to really help facilitate that, because that is what's the face of pulmonary hypertension is and that's what it's all about.

Moving forward, some of the focus we really want to do is looking at identifying kiddos and family members earlier on and using things that we have; these biorepositories and trying to identify what makes certain patients different than others and then really try to tailor therapy to get the best outcome for these kids. There is going to be a lot of excitement moving forward and the biggest thing will be that this should improve care for our kids.

Dr. Angela Bates is pediatric PH specialist from Stollery Children’s Hospital. She is committed to conducting research in pediatric patients in the areas of critical care, specifically those with congenital heart disease and those affected by infectious diseases in the critical care arena, as well as patients presenting with pulmonary hypertension of all etiologies. Her research is focused on improving diagnosis, treatment, outcomes and resources available to these patients. In this episode, Dr. Bates discusses the importance of early diagnosis.

I'm Dr. Angela Bates. I work at the Stollery Children's Hospital in Edmonton, Alberta, Canada. I take care of the pediatric pulmonary hypertension program there. I'm the medical lead, as well as I do pediatric cardiac ICU. So I see both inpatient and outpatient kiddos, all across Western Canada.

I think pulmonary hypertension in kids - the thing that sets it apart from other lung diseases is we talk about being subclinical, meaning a lot of these kids can live with this disease without having any symptoms. By the time they actually show up, they can be very sick. They can be as sick as being in the ICU. They can show even as sick as dying from this disease. We have a this window of opportunity to make a huge difference with all available drugs that we have now-a-days including oral medications which we didn't have 10 or 15 years ago.

The other thing that's really remarkable is that this is an amazing group of children that really can do a lot despite being quite sick, if you look at their echocardiograms and look at some of their other different investigations. That's kind of remarkable with these kids. Again, [it can] also kind of trick us practitioners in terms of how aggressive we are with treatment. I think sometimes we aren't thinking about screening them, we're not thinking doing an ECG or ECHO. They give us very few clues early on.

Also a lot of our gold standard of tests are invasive. The gold standard would be doing a cardiac catheterization which requires sedation, a general anesthetic. It requires actually putting catheters into the groin. Those are all things that sound scary to parents, scary to kids, and we respect the fact that some of these kiddos; they have a high risk of something going bad in these procedures. So I think it makes it a bit difficult. That being said, we have really come a long way in terms of noninvasive investigations. Our ECHO technology is better. We now have cardiac MRI and everyday people in the radiology world are looking at trying to find more reliable noninvasive tools.

For us the challenge is a few things. For one, our group of pediatric pulmonary hypertension is very diverse. So two big groups are very different. You have these arterial pulmonary hypertension kiddos. They can either be hereditary or idiopathic related heart disease or other things like connective tissue. Then you have another group of developmental lung diseases. Even [with] treatments. Are there certain treatments that are useful in one group versus another? Some of these kiddos are very little, looking at non-invasive techniques of diagnosing and following these kids is very challenging. That's really where a lot of our research is headed now-a-days.

Then just even clinical end points. Because it's hard to really identify what matters the most for these kids. Obviously, yes, survival matters, but is there something that we can follow as a marker that's a little bit more sensitive? That we can follow that will change the earlier on disease progression that will help us to modify the therapy. Then again another thing that's just a little bit challenging is even different drugs that are available. So, oral medication we do have a lot more. A lot of them aren't approved in pediatrics, so we certainly use lots of medications off label but even what's available for them to get at home. Whether it's subcutaneous delivered or IV and so forth. That's some of the challenges, but also exciting areas for us to move forward in PH. If you look back at the older literature, diagnosing kids late in the game - their prognosis was quite terrible. Median survival without treatment was around 18 months. Now that we know that introducing different therapies earlier on, these kiddos can live 10-15 years, which gives them that window that; one to obviously be kids and do well but, two to get to a point where maybe lung transplant is a better option than when they were 5 years of age. It also gives us more time to find better treatments. Certainly implementing therapies early on, not only improves their survival but just their quality of life which really is what's most important to parents, to kids, and as well as to us as health care professionals.

In terms of advice for a general practitioner or a pediatrician in the community following these kiddos is, even though it's a scary disease educating yourself is very important. Empowering the families, listening to the families. And then, always not being afraid to ask advice. We're never too busy to talk on the phone. We are never too busy to help follow these kids in the community with you, to see them in our clinic, to provide more education.

The biggest thing for these kiddos is quality of life. Although sometimes we may have to sort of negotiate what sort of therapies are best in these kids. Really in the end, these kiddos should be at home with their families, out doing activities that they enjoy. We work within those constraints but to truly focus on how can kids best live out their childhood.

If you have a kiddo that just doesn't fit the normal mold of what you're used to seeing for common childhood illnesses like asthma or that sort of thing, think about pulmonary hypertension. And if your tests don’t make sense, certainly think of pulmonary hypertension. And I think the other thing is you can do a simple cardiac exam. If you can feel a second heart sound that's abnormal, if the kiddo looks like there's a right heart failure, there's a reason for that. A simple thing like an ECG is a great place to start. There is a lot of things you can do in the office to screen these kiddos and then pick up the phone.

The field of pulmonary hypertension both in the pediatric and the adult world is always moving forward and we really try to empower our families to really help facilitate that, because that is what's the face of pulmonary hypertension is and that's what it's all about.

Moving forward, some of the focus we really want to do is looking at identifying kiddos and family members earlier on and using things that we have; these biorepositories and trying to identify what makes certain patients different than others and then really try to tailor therapy to get the best outcome for these kids. There is going to be a lot of excitement moving forward and the biggest thing will be that this should improve care for our kids.

]]>fullEpisode 214 - Angela Bates, MDAngela Bates, MD - Early Pulmonary Hypertension DiagnosisMon, 21 Jan 2019 13:00:00 +0000Dr. Angela Bates is pediatric PH specialist from Stollery Children’s Hospital. She is committed to conducting research in pediatric patients in the areas of critical care, specifically those with congenital heart disease and those affected by infectious diseases in the critical care arena, as well as patients presenting with pulmonary hypertension of all etiologies. Her research is focused on improving diagnosis, treatment, outcomes and resources available to these patients. In this episode, Dr. Bates discusses the importance of early diagnosis.

]]>Dr. Angela Bates is pediatric PH specialist from Stollery Children’s Hospital. She is committed to conducting research in pediatric patients in the areas of critical care, specifically those with congenital heart disease and those affected by infectious diseases in the critical care arena, as well as patients presenting with pulmonary hypertension of all etiologies. Her research is focused on improving diagnosis, treatment, outcomes and resources available to these patients. In this episode, Dr. Bates discusses the importance of early diagnosis.

Dr. John Granton the head of Respirology at University Health Network, Mount Sinai Hospital and Women’s College Hospital. He is a consultant in respirology and critical care at the Toronto General Hospital and Professor of Medicine at the University of Toronto. He established and remains the director of the pulmonary hypertension (PH) program at the University Health Network. In this episode, Dr. Granton discusses the importance of early diagnosis.

My name is John Granton. I'm a lung doctor working at the Toronto General Hospital, which is part of the University Health Network at the University of Toronto, and I specialize in critical care in pulmonary hypertension.

I first got into pulmonary hypertension really just at a meeting were one of the transplant doctors spoke to me and said, "You know, we know we need somebody to start looking after pulmonary hypertension patients. We have this new drug, Flolan coming down nobody's available to start using it or understanding it, and we need somebody to start work working with us, looking after these patients.” So I thought it was a neat opportunity so I took it on.

It's a disease of the blood vessels of the lungs and my analogy is like taking a garden hose and kinking the garden hose and as you know, the flow in the hose decreases, so the flow through the lung circulation decreases. And similar to if you're watering your garden or trying to wash your car, you can't get enough stream up through the hole so you can't do the things that you want to do. And the same for the patient. They can exercise and do things. If you look at the hose itself, it actually becomes pressurized and that's the pressure we're measuring. But is not the pressure that's the problem it’s the kinking that's the problem. As the kinking gets worse and worse and worse, the flow continues to go down, putting more pressure on the hose and stressing the hose which is the right side of the heart and the right side of the heart starts to fail as the kinking gets worse.

And so what we try to do and treat patients is relieve the kink. As a result, the pressures go down, the flow goes up and you can start to do what you want to do. You can walk further, enjoy life better, spend more time doing the things you enjoy.

Most people just come in because they feel little short of breath. If you're a family doctor, you have a lot of people coming in to see you because they feel short of breath. And so, it's really difficult to tease out just by looking at somebody that they have pulmonary hypertension, which is an uncommon condition compared to more common conditions. Even deconditioning, just being out of shape, which is probably a much more common problem that most North Americans face these days. But I think progression, particularly in a young person, which is out of character for them, you have to pay attention to. And doing the right tests. We often see people being prescribed inhalers, or puffers, to relieve asthma without formally testing for the presence of asthma. We recognize the importance of making the right to diagnosis and using the right treatment.

So doing breathing tests, if you don't find asthma then look at something else. Then as you work your way through the possibilities, consider doing an echocardiogram, maybe not looking for pulmonary hypertension, but seeing if they have a heart related cause for their difficulty breathing. By doing that as well, you can investigate the possibility of them having pulmonary hypertension, potentially as the cause why they feel short of breath. So I think it's thinking about it during the right test, making sure you try to find the diagnosis and then move on if it's not what you think it is.

So, the big red flags for us are when people start to notice swelling of their legs, as the fluid starts to build up. People start to feel faint or actually faint when they start to exercise. That really implies that there something more sinister going on and should really prompt a much more immediate investigation, really directed at the heart and the circulation in the broadest sense. Again the echocardiogram is a really useful tool to tease that out.

We certainly recognize there are people out there with conditions that are at higher risk for developing pulmonary hypertension like scleroderma, for example. We recognize the importance of asking questions. “Are you feeling more short of breath,” doing breathing test to see if there's a problem with the circulation and the lung. And also doing ultrasounds or echocardiograms to see if there is stress on the heart, and finding the disease earlier, and treating earlier.

First of all, pulmonary hypertension as I said is uncommon unlike asthma and COPD and emphysema, which is much more common. It affects the blood vessels unlike other conditions which affect the spongy bits of the lung. But they share commonalities or similarities in that a lot of people just feel short of breath. It distinguishes itself also by the treatments which are available, which are very different from other causes of lung disease.

If you're living in a downtown environment where you have access to specialists, you're probably diagnosed a little faster but we realize that not everybody is living in a downtown center and has access to specialists. I think as you work through the possibilities, that takes time and you go from one test to another, to another. Then the patient tries to get an appointment with a specialist. They in turn raise the possibility, do the echocardiogram, and then refer them on to another specialist who then refers to a PH center.

I think recognizing that if you really think it's there, contacting a pulmonary hypertension center to say, “Is this a possibility, and should this patient be seeing you in advance,” would be helpful to speed that up. I know all the pulmonary hypertension centers make themselves available to speak to primary care physicians and as well as specialists, internists, cardiologists, respirologists, rheumatologists to have that conversation.

By the time you actually present with having difficulty breathing and feeling unwell, you've lost a lot of your circulation and so it's already fairly late on in the condition. There's been a lot of damage already. We believe that earlier diagnosis and earlier treatment as a result, stands a better chance of actually working and improving the pressures. There's a notion that if you can reduce the pressures you may prevent some progression of the condition over time. So I think earlier intervention in most things makes sense and pulmonary hypertension is likely also the same sort of story that the earlier you find the condition, the earlier we treat it aggressively, the more likely the person is to have a better outcome.

I would appeal to the family doctors that when you see somebody with shortness of breath, do the right tests. If you're worried about asthma or lung disease, prove it before he just give them a prescription for inhalers. Do the breathing test to see if they have asthma or not and use the appropriate drugs if they do. If they don't, then you have to take the next step and figure out why their short of breath and that would shorten a lot, the delay in getting the appropriate referral to a center if they have pulmonary hypertension.

Dr. John Granton the head of Respirology at University Health Network, Mount Sinai Hospital and Women’s College Hospital. He is a consultant in respirology and critical care at the Toronto General Hospital and Professor of Medicine at the University of Toronto. He established and remains the director of the pulmonary hypertension (PH) program at the University Health Network. In this episode, Dr. Granton discusses the importance of early diagnosis.

My name is John Granton. I'm a lung doctor working at the Toronto General Hospital, which is part of the University Health Network at the University of Toronto, and I specialize in critical care in pulmonary hypertension.

I first got into pulmonary hypertension really just at a meeting were one of the transplant doctors spoke to me and said, "You know, we know we need somebody to start looking after pulmonary hypertension patients. We have this new drug, Flolan coming down nobody's available to start using it or understanding it, and we need somebody to start work working with us, looking after these patients.” So I thought it was a neat opportunity so I took it on.

It's a disease of the blood vessels of the lungs and my analogy is like taking a garden hose and kinking the garden hose and as you know, the flow in the hose decreases, so the flow through the lung circulation decreases. And similar to if you're watering your garden or trying to wash your car, you can't get enough stream up through the hole so you can't do the things that you want to do. And the same for the patient. They can exercise and do things. If you look at the hose itself, it actually becomes pressurized and that's the pressure we're measuring. But is not the pressure that's the problem it’s the kinking that's the problem. As the kinking gets worse and worse and worse, the flow continues to go down, putting more pressure on the hose and stressing the hose which is the right side of the heart and the right side of the heart starts to fail as the kinking gets worse.

And so what we try to do and treat patients is relieve the kink. As a result, the pressures go down, the flow goes up and you can start to do what you want to do. You can walk further, enjoy life better, spend more time doing the things you enjoy.

Most people just come in because they feel little short of breath. If you're a family doctor, you have a lot of people coming in to see you because they feel short of breath. And so, it's really difficult to tease out just by looking at somebody that they have pulmonary hypertension, which is an uncommon condition compared to more common conditions. Even deconditioning, just being out of shape, which is probably a much more common problem that most North Americans face these days. But I think progression, particularly in a young person, which is out of character for them, you have to pay attention to. And doing the right tests. We often see people being prescribed inhalers, or puffers, to relieve asthma without formally testing for the presence of asthma. We recognize the importance of making the right to diagnosis and using the right treatment.

So doing breathing tests, if you don't find asthma then look at something else. Then as you work your way through the possibilities, consider doing an echocardiogram, maybe not looking for pulmonary hypertension, but seeing if they have a heart related cause for their difficulty breathing. By doing that as well, you can investigate the possibility of them having pulmonary hypertension, potentially as the cause why they feel short of breath. So I think it's thinking about it during the right test, making sure you try to find the diagnosis and then move on if it's not what you think it is.

So, the big red flags for us are when people start to notice swelling of their legs, as the fluid starts to build up. People start to feel faint or actually faint when they start to exercise. That really implies that there something more sinister going on and should really prompt a much more immediate investigation, really directed at the heart and the circulation in the broadest sense. Again the echocardiogram is a really useful tool to tease that out.

We certainly recognize there are people out there with conditions that are at higher risk for developing pulmonary hypertension like scleroderma, for example. We recognize the importance of asking questions. “Are you feeling more short of breath,” doing breathing test to see if there's a problem with the circulation and the lung. And also doing ultrasounds or echocardiograms to see if there is stress on the heart, and finding the disease earlier, and treating earlier.

First of all, pulmonary hypertension as I said is uncommon unlike asthma and COPD and emphysema, which is much more common. It affects the blood vessels unlike other conditions which affect the spongy bits of the lung. But they share commonalities or similarities in that a lot of people just feel short of breath. It distinguishes itself also by the treatments which are available, which are very different from other causes of lung disease.

If you're living in a downtown environment where you have access to specialists, you're probably diagnosed a little faster but we realize that not everybody is living in a downtown center and has access to specialists. I think as you work through the possibilities, that takes time and you go from one test to another, to another. Then the patient tries to get an appointment with a specialist. They in turn raise the possibility, do the echocardiogram, and then refer them on to another specialist who then refers to a PH center.

I think recognizing that if you really think it's there, contacting a pulmonary hypertension center to say, “Is this a possibility, and should this patient be seeing you in advance,” would be helpful to speed that up. I know all the pulmonary hypertension centers make themselves available to speak to primary care physicians and as well as specialists, internists, cardiologists, respirologists, rheumatologists to have that conversation.

By the time you actually present with having difficulty breathing and feeling unwell, you've lost a lot of your circulation and so it's already fairly late on in the condition. There's been a lot of damage already. We believe that earlier diagnosis and earlier treatment as a result, stands a better chance of actually working and improving the pressures. There's a notion that if you can reduce the pressures you may prevent some progression of the condition over time. So I think earlier intervention in most things makes sense and pulmonary hypertension is likely also the same sort of story that the earlier you find the condition, the earlier we treat it aggressively, the more likely the person is to have a better outcome.

I would appeal to the family doctors that when you see somebody with shortness of breath, do the right tests. If you're worried about asthma or lung disease, prove it before he just give them a prescription for inhalers. Do the breathing test to see if they have asthma or not and use the appropriate drugs if they do. If they don't, then you have to take the next step and figure out why their short of breath and that would shorten a lot, the delay in getting the appropriate referral to a center if they have pulmonary hypertension.

]]>fullEpisode 213 - John Granton, MDJohn Granton, MD - Early Pulmonary Hypertension DiagnosisThu, 17 Jan 2019 13:00:00 +0000Dr. John Granton the head of Respirology at University Health Network, Mount Sinai Hospital and Women’s College Hospital. He is a consultant in respirology and critical care at the Toronto General Hospital and Professor of Medicine at the University of Toronto. He established and remains the director of the pulmonary hypertension (PH) program at the University Health Network. In this episode, Dr. Granton discusses the importance of early diagnosis.

]]>Dr. John Granton the head of Respirology at University Health Network, Mount Sinai Hospital and Women’s College Hospital. He is a consultant in respirology and critical care at the Toronto General Hospital and Professor of Medicine at the University of Toronto. He established and remains the director of the pulmonary hypertension (PH) program at the University Health Network. In this episode, Dr. Granton discusses the importance of early diagnosis.

]]>06:17cleanblood,canada,early,research,heart,awareness,asthma,iv,breathless,rare,ph,diagnosis,pulmonary,raredisease,catheterization,pha,clinicaltrials,pulmonaryhypertension,phaware,lungdiseaseDr. John Granton the head of Respirology at University Health Network, Mount Sinai Hospital and Women’s College Hospital. He is a consultant in respirology and critical care at the Toronto General Hospital and Professor of Medicine at the University of Toronto. He established and remains the director of the pulmonary hypertension (PH) program at the University Health Network. Learn more about pulmonary hypertension at www.phaware.global #phaware #phawareMD314fullphaware global associationSanjay Mehta, MD - phaware® interview 212Tue, 15 Jan 2019 13:00:00 +0000

Sanjay Mehta, MD, FRCPC, FCCP, isPHA Canada’sChair. He is additionally Professor of Medicine at the University of Western Ontario, and Director of the Southwest Ontario Pulmonary Hypertension Clinic at the London Health Sciences Center in London, Canada. He is also Chair of the Pulmonary Vascular Disease Committee of the Canadian Thoracic Society. Dr. Mehta has been caring for patients with PH and doing research in PH since 1989. In this episode, Dr. Mehta discusses the importance of early diagnosis.

I'm Dr. Sanjay Mehta. I'm a respirologist in London, Ontario, also a professor of medicine and most importantly I'm director of the Southwest Ontario Pulmonary Hypertension clinic and finally, but very importantly, I'm chair of Pulmonary Hypertension Association of Canada.

I've been looking after patients in the London clinic for 22 years, but I first got exposed and introduced to pulmonary hypertension 30 years ago as a medical resident at McGill University in Montreal. As a young physician, these were rare patients I didn't see very often, but I recognized they were very sick and needed our help. As well, I was quite interested in looking after them to learn more about them, their illness and especially the fascinating interaction of the heart and the lungs. Even then, I recognized that we could help these patients, and this is only still true today. They're very sick patients that need our help and they have much to teach all physicians.

Pulmonary hypertension is another form of hypertension or high blood pressure, but this time specifically in the blood vessels that feed the lungs or the pulmonary arteries. The most important issue is that the right heart has to pump blood to the lungs and it gets under strain and pulmonary hypertension. The most important complication is failure of the right ventricle or the right side of the heart. Patients are affected by symptoms like shortness of breath and fatigue and eventually as the disease progresses, they get dizziness, syncope, and chest pain. The most important thing to recognize is it's a serious illness. This is an illness that still kills people in two to three years if it's not diagnosed or treated and yet we do have many treatment options that help patients.

The biggest issue is that pulmonary hypertension is not being recognized or diagnosed soon enough and this is the biggest issue we hear from patients and their families, because they have symptoms for often two years before they get a diagnosis and finally get treated. I don't think this is an issue of awareness. Most doctors have heard of pulmonary hypertension. The challenges is this is an issue of ignorance, not knowing what the disease is, how to recognize and especially diagnose the disease and maybe most importantly these days not recognizing this as a treatable illness, that we have excellent therapies that are helping many patients with pulmonary hypertension.

It's very hard for a good general doctor to be able to recognize all serious heart and lung disease. They're expected to deal with common illnesses like Asthma and COPD and heart failure that cause people to be short of breath, very much like pulmonary hypertension. The challenge is to always be aware that there are uncommon illnesses that are always going to be presenting themselves and to think if you're treating somebody that's not getting better and not responding as you expect, could it be something less common? Then to know that the most important test for pulmonary hypertension is an echocardiogram. This will pick up pulmonary hypertension in most patients, especially when it's clinically significant or important. Pulmonary hypertension cannot easily be diagnosed on physical examination and the history is challenging, because patients have the same symptoms as many heart and lung conditions and so I always emphasize, think about it, and then an echocardiogram is the first and best test.

Pulmonary hypertension is a difficult diagnosis. The problem is that the symptoms patients experience, shortness of breath, tiredness, eventually syncope, passing out or chest pain are very similar with many other common lung and heart diseases. For example, patients with Asthma and COPD are always short of breath, and so it's difficult for a family doctor or a community specialist to always think about pulmonary hypertension. The only issue is that when you treat somebody for common issues and they don't get better, they don't respond - always be thinking, could I be missing something and that's where pulmonary hypertension comes in. It's easily missed especially because on physical examination it's not an easy diagnosis to pick up and we don't recommend looking for it intensively. The most important thing is think about it and then an echocardiogram is the right first test, as it picks up pulmonary hypertension in most patients, especially if the disease is clinically important or significant.

The diagnosis of pulmonary hypertension is most challenging because it takes a little bit of a framework to understand what are the possible causes. I think doctors understand that you can get pulmonary hypertension if you have heart disease or lung disease or very importantly pulmonary thrombo embolism or pulmonary emboli, and so it takes a very clear workup where you look for those conditions, heart disease, lung disease, and blood clots, and recognize that many of those can contribute to pulmonary hypertension. The most important thing is to think about addressing each of those points. If you treat all those conditions, that's what it takes to improve a patient with pulmonary hypertension.

For the average family physician or community specialist who has a patient that's been diagnosed with pulmonary hypertension, I would strongly advise you to learn from your patient. Understand their illness, understand their symptoms and their limitations. As well, they're being cared for often in a PH expert center, and so learn from them. They'll help you understand what tests are important to diagnose pulmonary hypertension in your future patients, as well as what tests are useful to follow your patient with pulmonary hypertension, and then also what treatment options there are.

This is an illness that fortunately patients are better, they're living longer, and so a family doctor is so important because there are many other things that have to be looked after besides just the pulmonary hypertension. These patients need anticoagulation. They need diuretics to control their fluid and a family doctor can very well look after that. As well as patients live longer, they need the usual cancer screening, they need their diabetes and their heartburn looked after and their arthritis. There's many roles for a family physician to do a good job looking after pulmonary hypertension patients and to along the way, learn about pulmonary hypertension to diagnose the next patient.

It's been quite an honor to be involved in pulmonary hypertension and learn about it and look after patients for almost 30 years. It's also been an incredible time because the pace of development of research, knowledge and diagnostic approaches and treatments is almost unparalleled in any field of medicine. From an illness that used to kill everybody within two to three years without any treatment options, we're now at a stage that we can reliably offer patients a quality of life that's better and longer survival. Average seven to 10 years, but many patients now living 20 years and longer with pulmonary hypertension. So all of that's changed dramatically because of great science and research and it's a wonderful journey to be part of along with patients and their families.

Physicians in almost any branch of medicine will run into these patients. They present to family doctors. They present to emergency rooms, they present the specialist all the time, because they get many complications of their disease if they're not diagnosed and the most important thing to recognize is they need a diagnosis because you can help them. We can help them with the wonderful treatment options we have, medical and surgical. These have been shown to improve their quality of life, reduce their symptoms, improve their functional exercise capacity, and very importantly, live longer and better lives.

Sanjay Mehta, MD, FRCPC, FCCP, isPHA Canada’sChair. He is additionally Professor of Medicine at the University of Western Ontario, and Director of the Southwest Ontario Pulmonary Hypertension Clinic at the London Health Sciences Center in London, Canada. He is also Chair of the Pulmonary Vascular Disease Committee of the Canadian Thoracic Society. Dr. Mehta has been caring for patients with PH and doing research in PH since 1989. In this episode, Dr. Mehta discusses the importance of early diagnosis.

I'm Dr. Sanjay Mehta. I'm a respirologist in London, Ontario, also a professor of medicine and most importantly I'm director of the Southwest Ontario Pulmonary Hypertension clinic and finally, but very importantly, I'm chair of Pulmonary Hypertension Association of Canada.

I've been looking after patients in the London clinic for 22 years, but I first got exposed and introduced to pulmonary hypertension 30 years ago as a medical resident at McGill University in Montreal. As a young physician, these were rare patients I didn't see very often, but I recognized they were very sick and needed our help. As well, I was quite interested in looking after them to learn more about them, their illness and especially the fascinating interaction of the heart and the lungs. Even then, I recognized that we could help these patients, and this is only still true today. They're very sick patients that need our help and they have much to teach all physicians.

Pulmonary hypertension is another form of hypertension or high blood pressure, but this time specifically in the blood vessels that feed the lungs or the pulmonary arteries. The most important issue is that the right heart has to pump blood to the lungs and it gets under strain and pulmonary hypertension. The most important complication is failure of the right ventricle or the right side of the heart. Patients are affected by symptoms like shortness of breath and fatigue and eventually as the disease progresses, they get dizziness, syncope, and chest pain. The most important thing to recognize is it's a serious illness. This is an illness that still kills people in two to three years if it's not diagnosed or treated and yet we do have many treatment options that help patients.

The biggest issue is that pulmonary hypertension is not being recognized or diagnosed soon enough and this is the biggest issue we hear from patients and their families, because they have symptoms for often two years before they get a diagnosis and finally get treated. I don't think this is an issue of awareness. Most doctors have heard of pulmonary hypertension. The challenges is this is an issue of ignorance, not knowing what the disease is, how to recognize and especially diagnose the disease and maybe most importantly these days not recognizing this as a treatable illness, that we have excellent therapies that are helping many patients with pulmonary hypertension.

It's very hard for a good general doctor to be able to recognize all serious heart and lung disease. They're expected to deal with common illnesses like Asthma and COPD and heart failure that cause people to be short of breath, very much like pulmonary hypertension. The challenge is to always be aware that there are uncommon illnesses that are always going to be presenting themselves and to think if you're treating somebody that's not getting better and not responding as you expect, could it be something less common? Then to know that the most important test for pulmonary hypertension is an echocardiogram. This will pick up pulmonary hypertension in most patients, especially when it's clinically significant or important. Pulmonary hypertension cannot easily be diagnosed on physical examination and the history is challenging, because patients have the same symptoms as many heart and lung conditions and so I always emphasize, think about it, and then an echocardiogram is the first and best test.

Pulmonary hypertension is a difficult diagnosis. The problem is that the symptoms patients experience, shortness of breath, tiredness, eventually syncope, passing out or chest pain are very similar with many other common lung and heart diseases. For example, patients with Asthma and COPD are always short of breath, and so it's difficult for a family doctor or a community specialist to always think about pulmonary hypertension. The only issue is that when you treat somebody for common issues and they don't get better, they don't respond - always be thinking, could I be missing something and that's where pulmonary hypertension comes in. It's easily missed especially because on physical examination it's not an easy diagnosis to pick up and we don't recommend looking for it intensively. The most important thing is think about it and then an echocardiogram is the right first test, as it picks up pulmonary hypertension in most patients, especially if the disease is clinically important or significant.

The diagnosis of pulmonary hypertension is most challenging because it takes a little bit of a framework to understand what are the possible causes. I think doctors understand that you can get pulmonary hypertension if you have heart disease or lung disease or very importantly pulmonary thrombo embolism or pulmonary emboli, and so it takes a very clear workup where you look for those conditions, heart disease, lung disease, and blood clots, and recognize that many of those can contribute to pulmonary hypertension. The most important thing is to think about addressing each of those points. If you treat all those conditions, that's what it takes to improve a patient with pulmonary hypertension.

For the average family physician or community specialist who has a patient that's been diagnosed with pulmonary hypertension, I would strongly advise you to learn from your patient. Understand their illness, understand their symptoms and their limitations. As well, they're being cared for often in a PH expert center, and so learn from them. They'll help you understand what tests are important to diagnose pulmonary hypertension in your future patients, as well as what tests are useful to follow your patient with pulmonary hypertension, and then also what treatment options there are.

This is an illness that fortunately patients are better, they're living longer, and so a family doctor is so important because there are many other things that have to be looked after besides just the pulmonary hypertension. These patients need anticoagulation. They need diuretics to control their fluid and a family doctor can very well look after that. As well as patients live longer, they need the usual cancer screening, they need their diabetes and their heartburn looked after and their arthritis. There's many roles for a family physician to do a good job looking after pulmonary hypertension patients and to along the way, learn about pulmonary hypertension to diagnose the next patient.

It's been quite an honor to be involved in pulmonary hypertension and learn about it and look after patients for almost 30 years. It's also been an incredible time because the pace of development of research, knowledge and diagnostic approaches and treatments is almost unparalleled in any field of medicine. From an illness that used to kill everybody within two to three years without any treatment options, we're now at a stage that we can reliably offer patients a quality of life that's better and longer survival. Average seven to 10 years, but many patients now living 20 years and longer with pulmonary hypertension. So all of that's changed dramatically because of great science and research and it's a wonderful journey to be part of along with patients and their families.

Physicians in almost any branch of medicine will run into these patients. They present to family doctors. They present to emergency rooms, they present the specialist all the time, because they get many complications of their disease if they're not diagnosed and the most important thing to recognize is they need a diagnosis because you can help them. We can help them with the wonderful treatment options we have, medical and surgical. These have been shown to improve their quality of life, reduce their symptoms, improve their functional exercise capacity, and very importantly, live longer and better lives.

]]>fullEpisode 212 - Sanjay Mehta, MDSanjay Mehta, MD - Early Pulmonary Hypertension DiagnosisMon, 14 Jan 2019 13:00:00 +0000Sanjay Mehta, MD, FRCPC, FCCP, isPHA Canada’sChair. He is additionally Professor of Medicine at the University of Western Ontario, and Director of the Southwest Ontario Pulmonary Hypertension Clinic at the London Health Sciences Center in London, Canada. He is also Chair of the Pulmonary Vascular Disease Committee of the Canadian Thoracic Society. Dr. Mehta has been caring for patients with PH and doing research in PH since 1989. In this episode, Dr. Mehta discusses the importance of early diagnosis.

]]>Sanjay Mehta, MD, FRCPC, FCCP, isPHA Canada’sChair. He is additionally Professor of Medicine at the University of Western Ontario, and Director of the Southwest Ontario Pulmonary Hypertension Clinic at the London Health Sciences Center in London, Canada. He is also Chair of the Pulmonary Vascular Disease Committee of the Canadian Thoracic Society. Dr. Mehta has been caring for patients with PH and doing research in PH since 1989. In this episode, Dr. Mehta discusses the importance of early diagnosis.

]]>07:10cleanblood,canada,early,research,heart,awareness,asthma,iv,breathless,rare,ph,diagnosis,pulmonary,raredisease,catheterization,pha,clinicaltrials,pulmonaryhypertension,phaware,lungdiseaseSanjay Mehta, MD, FRCPC, FCCP, is PHA Canada’s Chair. He is additionally Professor of Medicine at the University of Western Ontario, and Director of the Southwest Ontario Pulmonary Hypertension Clinic at the London Health Sciences Center in London, Canada. Dr. Mehta has been caring for patients with PH and doing research in PH since 1989. In this episodes, Dr. Mehta discusses the importance of early diagnosis. Learn more about pulmonary hypertension at www.phaware.global #phaware #phawareMD
313fullphaware global associationLori Heaton - phaware® interview 211Fri, 30 Nov 2018 13:00:00 +0000

My name is Lori Heaton. I was a parent caregiver to my daughter Madison Heaton. We are from Provo, Utah. Maddie was diagnosed with pulmonary hypertension in 1997.

After we found out that she had an ASD, we got treatment at Primary Children's Hospital in Salt Lake City, Utah, but her primary cardiologist was Dr. Ivy at Children's Hospital Colorado in Denver. It was 1997, and so when we got the diagnosis we were told to go home and make her comfortable for whatever time she had left, because there were no good options for treatment for pulmonary hypertension other than lung transplant and they didn't recommend it in a four year old. That's why we were told just to go home and make her comfortable.

When we found out about Dr. Ivy. His program was only two years in the making when we came along. She was placed on IV Flolan (epoprostenol) immediately, because her pressures were really high. Any oral medications would've been ineffective for her advanced disease. Maddie, I felt was kind of one of the early pioneers in testing out these new therapies. Whenever any new treatment got approved, she would try it. So she went from IV Flolan to IV Remodulin (treprostinil), but the problem was she just would get a lot of line infections.

In 1999, we found out that she had leukemia. They recommended chemotherapy, but they had their reservations, because some of the chemotherapy drugs are hard on your heart. Since her heart function was compromised from the pulmonary hypertension, they were reluctant to give her any chemotherapy. We wanted Maddie to have every chance to live. Again, we made a hard decision to put her on the chemotherapy.

How chemotherapy works with leukemia is they do a bone marrow biopsy and they see how many leukemia cells are in the bone marrow. Her bone marrow, I think was impacted like 95% with leukemia cells. Immediately, they started her on very aggressive chemotherapy, and within three weeks she was in remission with no leukemia detected. She went through the 18 months of chemotherapy and she did pretty well. The whole time, she was battling pulmonary hypertension too and like I said, struggling with a lot of IV line infections which every parent knows is a big deal and it's scary.

So when Ventavis (iloprost), the new inhaled therapy came along, she tried that. This was about when she was in middle school when she started that and she was trying to go to school, which is always a challenge with kids with pulmonary hypertension, because they can feel fine for a couple of weeks and then be out for three weeks because they're sick. Or they just don't have the energy to go full time. In the '90s, homeschooling wasn't as popular as it is today. And there wasn't a lot of options for homeschooling as there is today, so that was always a challenge.

She started on the Ventavis when she was in middle school. You have to nebulize like five times a day. And it can take anywhere between ten to 20 minutes. So she would always have to schedule time in between classes, during classes, any activity had to be interrupted by her doing her nebulizer treatments. She had really hard time complying with the schedule. It was just hard. She started to feel as much tethered to her nebulizer as she did with the permanent IV and so she did that for a couple of years and then decided to go on the subcutaneous Remodulin (treprostinil). She was always kind of reluctant to do that as well, because of the site pain that we had heard was so severe. She did that for about five years.

By this time, she was a young adult. As everyone knows pulmonary hypertension is a progressive disease. So by the time she was 20 years old, she had pretty much run out of all the options for treatment. She had gone through all the oral medications, she had gone through the IV medications and the inhaled and basically she was looking at a lung transplant for her only option and that was scary. I knew that it would be trading old problems for new problems. I was just hoping that a cure would come before we were faced with a lung transplant, but here we were.

We met with the transplant people at the University of Utah. At that time, Maddie had transferred from Dr. Ivy's care to Dr. Elliott's care, who's an adult pulmonary hypertension specialist in Salt Lake. He really recommended that we go find out about lung transplantation, and he said "I'll refer you anywhere you want to go, but the most important thing is family support. You need to have that big family support wherever you choose to go." Since we are all here, we chose the University of Utah and we were really impressed with their program.

So they did all the testing. They had to determine whether a heart lung transplant would be the best thing for Maddie or just repairing the hole and doing a bilateral lung transplant. She went through all the testing, they came back and decided that they could just repair that hole and then do a bilateral lung transplant, and then she got listed. Maddie and her younger sister were living down in St. George which is in southern Utah. It's about a 300 mile trip up to Salt Lake, so Maddie had to relocate. But she had an older sister living in Park City, which is only a 20 minute drive to the University of Utah, so she moved in with my daughter Janae while she got listed.

I think she was on the list about 42 days when she got the call. They have you come in and then you have to wait because they have to get the lungs from wherever they're coming from and then they have to make sure that the lungs are viable enough for them to be transplanted. If they're not, then you are sent home and you get put back on the list. It's called a dry run and it happens a lot. When she got the call, it was like five in the morning. She was up here and we were down in St. George and they said don't come up until we know it's a go. My husband Randy and I were just sitting there waiting on pins and needles until we heard that it was a go. Then it was and they wheeled her into the operating room while we were still on the road.

We got there and about six hours later the thoracic surgeon came out and said she did really well and she was only on a ventilator like six hours. She spent about two weeks in the hospital which is about average. It's hard because it's a big surgery with a lot of pain and then they put you on anti-rejection drugs and then they have to titrate them so that the dose is enough to suppress the immune system from rejecting the new lungs, but not enough that it puts her at risk for infection. It's a process and you don't feel well, because these are really heavy duty drugs. You adjust and you get better, but the thing is, back when she was little, I thought I will never put her through a transplant. But you know what, it was her decision, of course, because she was an adult by the time.

These kids, they want to live. They just want live and they want to have a normal life and she was able to have a good life. She was able to live an active life and not be tethered to an IV. And she could breathe. It's huge. But like I said, the anti-rejection drugs put you at a really high risk for an infection, any type of infection. She got a fungal infection in her lungs. Fungus, they're just really common. We breathe in fungal spores every day. People with regular immune systems it's no big deal. But people with weakened immune systems, it can make you sick. She eventually kind of went into chronic rejection, which is just scarring of those small bronchioles in the lungs.

She ended up in the hospital again. They put her eventually on life support. I remember the day that they admitted her. The doctor said, "you may not make it long enough to get another lung transplant and so I need to have you sign a living will." I think up until that point that was like the hardest thing I'd ever had to do. What's a living will is her wishes in writing so that we know what she wants us to do if she can't speak for herself. Eventually, she was put on life support and the thing about life support, the ventilator breathes for you but it doesn't treat the underlying disease and so her lungs were still progressively getting worse. Just like the doctor said, she wasn't going to make it long enough to get a lung transplant. But then she did.

It was a miracle that she was able to get a donor while she was still in the hospital. They urgently rushed her into surgery, and she was so sick from being on life support that giving her another double lung transplant was too risky. She wouldn't have survived the operation. So they gave her a single lung transplant. They told us that people can live a fairly normal life with one lung.

She did well with that and the recovery was quite a bit longer this time just because she was so sick going in that she kind of had to dig out of a hole basically. But she did, she did really well and again she was able to go to college and she was thinking about what she wanted to do with her life and what career options were available to her. She was literally given another chance to live. She wanted to travel. I always thought, even when she was little, that she knew that she wouldn't be here on this earth very long. She wanted to experience life.

Her and her sisters are very close and so they traveled around a lot. It was a few months before she passed away, she found some really cheap tickets to Amsterdam and she had always wanted to go there. So she booked the flight, and planned the trip and she went with her sisters and some friends and it was a really amazing trip for her, but she got sick over there. In her mind, it was just a cold. The transplant doctors had given their blessing for her to go, but they gave her some antibiotics and some steroids and things just in case. She got a respiratory infection basically there and she took the meds that were given to her and by the time she got home she was feeling fine. But then shortly after she just started getting progressively more short of breath and she was diagnosed with having another episode of rejection.

The doctor wanted to do a bronchoscopy to look down into her lungs to see what was going on. So in June, she had another bronchoscopy done. What happens with chronic rejection, those small airways scar, so you hold on to the Co2 which is the bad stuff that you're supposed to blow off. Well, she wasn't able to. She wasn't able to get rid of the Co2 and so it built up in her blood to where she went into respiratory arrest right after the bronchoscopy and went on life support. Basically, she couldn't come off because she only had one lung, and it was bad. Basically, there was no other choice for her. So we had to make the really hard decision to take her off life support. On June 21st of 2017 we had her removed from life support.

I had started writing a book even before she passed away, because even though this is a story that doesn't have a very happy ending, there were some really good things that I learned from being Maddie's caregiver all those years. I just wanted people to know that, you can go through hard things and still come out okay on the other end. You can learn good things. It's a book that just chronicles Maddie's life through pulmonary hypertension and the things that she taught me. It's about all the things that you can learn from going through hard things. It's for anyone who wants to become a better person through the adversity that they've gone through.

Each chapter focuses on a blessing that I had learned. One is faith or perseverance or living in the moment or being an advocate or learning to be happy. Things like that. It was something that I had thought about for years and years, but the timing just had to be right. The timing was right now. It is called Difficult Blessings. You can find it on Amazon and on Barnes & Noble. If any of you are a friend of mine on social media, if you want to contact me directly, I can actually even sign a copy for you.

So it's been a little over a year and I'm a lot better than I was last year, but I think about her every day. It's like a part of you is missing. But the thought of her being able to breathe again, it's comforting. She went through a lot in her life, but she was always happy. She never complained. She lived most of her life on one type of invasive therapy or another. But you learn to adapt and you learn to make these kids happy and try to give them a normal life. It’s a tricky thing trying to give someone a normal life who's on an IV 24/7. It's a challenge but you do. And there's thousands and thousands of parent caregivers that are doing that.

My name is Lori Heaton. I am aware that my daughter Madison Heaton was rare.

My name is Lori Heaton. I was a parent caregiver to my daughter Madison Heaton. We are from Provo, Utah. Maddie was diagnosed with pulmonary hypertension in 1997.

After we found out that she had an ASD, we got treatment at Primary Children's Hospital in Salt Lake City, Utah, but her primary cardiologist was Dr. Ivy at Children's Hospital Colorado in Denver. It was 1997, and so when we got the diagnosis we were told to go home and make her comfortable for whatever time she had left, because there were no good options for treatment for pulmonary hypertension other than lung transplant and they didn't recommend it in a four year old. That's why we were told just to go home and make her comfortable.

When we found out about Dr. Ivy. His program was only two years in the making when we came along. She was placed on IV Flolan (epoprostenol) immediately, because her pressures were really high. Any oral medications would've been ineffective for her advanced disease. Maddie, I felt was kind of one of the early pioneers in testing out these new therapies. Whenever any new treatment got approved, she would try it. So she went from IV Flolan to IV Remodulin (treprostinil), but the problem was she just would get a lot of line infections.

In 1999, we found out that she had leukemia. They recommended chemotherapy, but they had their reservations, because some of the chemotherapy drugs are hard on your heart. Since her heart function was compromised from the pulmonary hypertension, they were reluctant to give her any chemotherapy. We wanted Maddie to have every chance to live. Again, we made a hard decision to put her on the chemotherapy.

How chemotherapy works with leukemia is they do a bone marrow biopsy and they see how many leukemia cells are in the bone marrow. Her bone marrow, I think was impacted like 95% with leukemia cells. Immediately, they started her on very aggressive chemotherapy, and within three weeks she was in remission with no leukemia detected. She went through the 18 months of chemotherapy and she did pretty well. The whole time, she was battling pulmonary hypertension too and like I said, struggling with a lot of IV line infections which every parent knows is a big deal and it's scary. So when Ventavis (iloprost), the new inhaled therapy came along, she tried that. This was about when she was in middle school when she started that and she was trying to go to school, which is always a challenge with kids with pulmonary hypertension, because they can feel fine for a couple of weeks and then be out for three weeks because they're sick. Or they just don't have the energy to go full time. In the '90s, homeschooling wasn't as popular as it is today. And there wasn't a lot of options for homeschooling as there is today, so that was always a challenge.

She started on the Ventavis when she was in middle school. You have to nebulize like five times a day. And it can take anywhere between ten to 20 minutes. So she would always have to schedule time in between classes, during classes, any activity had to be interrupted by her doing her nebulizer treatments. She had really hard time complying with the schedule. It was just hard. She started to feel as much tethered to her nebulizer as she did with the permanent IV and so she did that for a couple of years and then decided to go on the subcutaneous Remodulin (treprostinil). She was always kind of reluctant to do that as well, because of the site pain that we had heard was so severe. She did that for about five years.

By this time, she was a young adult. As everyone knows pulmonary hypertension is a progressive disease. So by the time she was 20 years old, she had pretty much run out of all the options for treatment. She had gone through all the oral medications, she had gone through the IV medications and the inhaled and basically she was looking at a lung transplant for her only option and that was scary. I knew that it would be trading old problems for new problems. I was just hoping that a cure would come before we were faced with a lung transplant, but here we were.

We met with the transplant people at the University of Utah. At that time, Maddie had transferred from Dr. Ivy's care to Dr. Elliott's care, who's an adult pulmonary hypertension specialist in Salt Lake. He really recommended that we go find out about lung transplantation, and he said "I'll refer you anywhere you want to go, but the most important thing is family support. You need to have that big family support wherever you choose to go." Since we are all here, we chose the University of Utah and we were really impressed with their program.

So they did all the testing. They had to determine whether a heart lung transplant would be the best thing for Maddie or just repairing the hole and doing a bilateral lung transplant. She went through all the testing, they came back and decided that they could just repair that hole and then do a bilateral lung transplant, and then she got listed. Maddie and her younger sister were living down in St. George which is in southern Utah. It's about a 300 mile trip up to Salt Lake, so Maddie had to relocate. But she had an older sister living in Park City, which is only a 20 minute drive to the University of Utah, so she moved in with my daughter Janae while she got listed.

I think she was on the list about 42 days when she got the call. They have you come in and then you have to wait because they have to get the lungs from wherever they're coming from and then they have to make sure that the lungs are viable enough for them to be transplanted. If they're not, then you are sent home and you get put back on the list. It's called a dry run and it happens a lot. When she got the call, it was like five in the morning. She was up here and we were down in St. George and they said don't come up until we know it's a go. My husband Randy and I were just sitting there waiting on pins and needles until we heard that it was a go. Then it was and they wheeled her into the operating room while we were still on the road.

We got there and about six hours later the thoracic surgeon came out and said she did really well and she was only on a ventilator like six hours. She spent about two weeks in the hospital which is about average. It's hard because it's a big surgery with a lot of pain and then they put you on anti-rejection drugs and then they have to titrate them so that the dose is enough to suppress the immune system from rejecting the new lungs, but not enough that it puts her at risk for infection. It's a process and you don't feel well, because these are really heavy duty drugs. You adjust and you get better, but the thing is, back when she was little, I thought I will never put her through a transplant. But you know what, it was her decision, of course, because she was an adult by the time.

These kids, they want to live. They just want live and they want to have a normal life and she was able to have a good life. She was able to live an active life and not be tethered to an IV. And she could breathe. It's huge. But like I said, the anti-rejection drugs put you at a really high risk for an infection, any type of infection. She got a fungal infection in her lungs. Fungus, they're just really common. We breathe in fungal spores every day. People with regular immune systems it's no big deal. But people with weakened immune systems, it can make you sick. She eventually kind of went into chronic rejection, which is just scarring of those small bronchioles in the lungs.

She ended up in the hospital again. They put her eventually on life support. I remember the day that they admitted her. The doctor said, "you may not make it long enough to get another lung transplant and so I need to have you sign a living will." I think up until that point that was like the hardest thing I'd ever had to do. What's a living will is her wishes in writing so that we know what she wants us to do if she can't speak for herself. Eventually, she was put on life support and the thing about life support, the ventilator breathes for you but it doesn't treat the underlying disease and so her lungs were still progressively getting worse. Just like the doctor said, she wasn't going to make it long enough to get a lung transplant. But then she did.

It was a miracle that she was able to get a donor while she was still in the hospital. They urgently rushed her into surgery, and she was so sick from being on life support that giving her another double lung transplant was too risky. She wouldn't have survived the operation. So they gave her a single lung transplant. They told us that people can live a fairly normal life with one lung.

She did well with that and the recovery was quite a bit longer this time just because she was so sick going in that she kind of had to dig out of a hole basically. But she did, she did really well and again she was able to go to college and she was thinking about what she wanted to do with her life and what career options were available to her. She was literally given another chance to live. She wanted to travel. I always thought, even when she was little, that she knew that she wouldn't be here on this earth very long. She wanted to experience life.

Her and her sisters are very close and so they traveled around a lot. It was a few months before she passed away, she found some really cheap tickets to Amsterdam and she had always wanted to go there. So she booked the flight, and planned the trip and she went with her sisters and some friends and it was a really amazing trip for her, but she got sick over there. In her mind, it was just a cold. The transplant doctors had given their blessing for her to go, but they gave her some antibiotics and some steroids and things just in case. She got a respiratory infection basically there and she took the meds that were given to her and by the time she got home she was feeling fine. But then shortly after she just started getting progressively more short of breath and she was diagnosed with having another episode of rejection.

The doctor wanted to do a bronchoscopy to look down into her lungs to see what was going on. So in June, she had another bronchoscopy done. What happens with chronic rejection, those small airways scar, so you hold on to the Co2 which is the bad stuff that you're supposed to blow off. Well, she wasn't able to. She wasn't able to get rid of the Co2 and so it built up in her blood to where she went into respiratory arrest right after the bronchoscopy and went on life support. Basically, she couldn't come off because she only had one lung, and it was bad. Basically, there was no other choice for her. So we had to make the really hard decision to take her off life support. On June 21st of 2017 we had her removed from life support.

I had started writing a book even before she passed away, because even though this is a story that doesn't have a very happy ending, there were some really good things that I learned from being Maddie's caregiver all those years. I just wanted people to know that, you can go through hard things and still come out okay on the other end. You can learn good things. It's a book that just chronicles Maddie's life through pulmonary hypertension and the things that she taught me. It's about all the things that you can learn from going through hard things. It's for anyone who wants to become a better person through the adversity that they've gone through.

Each chapter focuses on a blessing that I had learned. One is faith or perseverance or living in the moment or being an advocate or learning to be happy. Things like that. It was something that I had thought about for years and years, but the timing just had to be right. The timing was right now. It is called Difficult Blessings. You can find it on Amazon and on Barnes & Noble. If any of you are a friend of mine on social media, if you want to contact me directly, I can actually even sign a copy for you. So it's been a little over a year and I'm a lot better than I was last year, but I think about her every day. It's like a part of you is missing. But the thought of her being able to breathe again, it's comforting. She went through a lot in her life, but she was always happy. She never complained. She lived most of her life on one type of invasive therapy or another. But you learn to adapt and you learn to make these kids happy and try to give them a normal life. It’s a tricky thing trying to give someone a normal life who's on an IV 24/7. It's a challenge but you do. And there's thousands and thousands of parent caregivers that are doing that.

My name is Lori Heaton. I am aware that my daughter Madison Heaton was rare.

]]>13:27cleanblood,research,heart,awareness,asthma,iv,breathless,rare,ph,transplant,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdisease“Even though this is a story that doesn't have a very happy ending, there were some really good things that I learned from being Maddie's caregiver all those years.” PH caregiver and author, Lori Heaton
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin #phaware #YoungWithPAH #ClinicalTrials @antidote_me 312fullphaware global associationKristine Ritchie - phaware® interview 210Tue, 27 Nov 2018 13:00:00 +0000

Pediatric PH caregiver Kristine Ritchie discusses her son Brendan’s pulmonary hypertension diagnosis. Kristine talks about the importance of advocating for her child, the power of online support groups and struggle of navigating a new normal.

My name is Kristine Ritchie, I am from the Central Interior of BC in West Kelowna. I am the parent of an idiopathic pulmonary hypertension child who is 10 years old, and he was diagnosed at four and a half years old on December 12th of 2012.

Brendan has always been a small child and we thought that was pretty normal. One day we decided to go for a walk in November, just up the road from our house. It wasn't a strenuous walk and it was probably about 10 o'clock in the morning. We walked across the street and probably about 500 meters up the path. Brendan said to his dad, "Daddy I'm tired." He let out a whimper and then he fell to the ground. He basically had a seizure. His eyes rolled back in his head, he couldn't talk, he didn't really know where he was. That started our journey.

We took him to our local hospital, Kelowna General Hospital and we waited for the pediatrician on call, because he wanted to come check him out. About two, two and a half weeks later, we had an echocardiogram and we got the results that he had pulmonary hypertension. We were flown down to Vancouver two days later and we stayed at BC Children's for nine days with a myriad of tests and now we actually travel from Kelowna to Edmonton to his PH specialist.

With the medication, they knew what they wanted to start him on. He started on sildenafil and because he was only four and a half years old, he didn't know how to swallow any pills. We were able to compound it. However, our insurance companies would not cover the medication due to the type of medication that it was. Our insurance company wouldn't cover erectile dysfunction drugs, so they refused the medication for him. It took three months basically of full-time letter writing and advocacy and fighting to get the medication covered from our insurance.

Basically, I was reaching out to anyone. I wanted to do anything I could for my child. When he was first diagnosed in Kelowna the pediatrician said, "Don't google pulmonary hypertension." The first thing I did that night after all the lights had gone off was google pulmonary hypertension. I figured out what it was and I just sat in the hospital room just not knowing what to do.

When we went to Vancouver, we had a great medical team and I'll [always] remember Dr. Sanatani. Although we don't get followed by him anymore, I will always remember he said that if I didn't advocate for my son, no one else would and I've remember that phrase for the last almost six years.

So I wrote letters to insurance, I wrote letters to my human resources. In the past six years I've had write letters throughout a myriad of circumstances to various levels within our school, our school district, our school board, the Minister of Education for BC. Just for help on so many different things that it has at some points been more than a full-time job advocating for my child.

My saving grace basically is a Facebook group: (Families of children with Pulmonary Hypertension). In the Central Okanagan, the Interior, there aren't hardly any other families that have children with pulmonary hypertension. Social media has been a great resource for me. I know not everyone is into social media, but the amount of people that you can connect with and everyone has a story, and everyone has been so uplifting and always there. Because it's all over the world, if you ask a question in the middle of the night, someone is going to be awake somewhere to help you answer your question and be a support for you. I feel like social media has been second to none for a caregiver such as myself.

Then also trying to get involved to your comfort level. Going and meeting other people. Reaching out to not only meet them, but listening to other people's stories and learning from other people, because so many people have traveled this road before us. If you can ask questions and follow the advice of others and the stories and the situations that others have been through, I think that it empowers you as a parent. If you're a patient and you find the patient support groups, then that I can empower you as a patient as well.

A lot of people say that it's creating a new normal and at the beginning there's so much going on, but just knowing that a new normal will eventually come down the road, sometimes it might take a few months. Right now, we are adjusting to the life of a subcutaneous pump, so still learning how to see my son attached to tubing and attached to a pump is still not quite normal yet. I know it's going to get there, but that's my biggest thing is a new normal will come, just give yourself time to adjust to everything that's going on.

Kids are incredibly resilient and resourceful. My son has barely complained even after a site change when sometimes he can't feed himself or do up his seat belt or he needs help getting dressed. He doesn't complain. We did have to work on him getting wet and into a pool, so just watching him be so proud of himself and take ownership and helping him advocate for himself. I think we can learn a lot from kids just watching them and how happy they are and how adaptive they are to everything.

Probably the only other thing that I would add, if I can do a broad spectrum… I think for kids, (because of course I have a pediatric PH patient), getting kids to meet other kids like themselves. Going to the Painted Turtle Camp if you can, talking to other people in Clinic Day because a lot of them are there for the same reason you are. Brendan thrives on meeting other kids and knowing their names and what have they got and it really helped him being able to see other children on a pump before him or seeing what they can do. Now he can share that he still goes tubing behind a boat, he goes golfing, he plays baseball and all of this is with an IV in his arm and a pump around his stomach.

Pediatric PH caregiver Kristine Ritchie discusses her son Brendan’s pulmonary hypertension diagnosis. Kristine talks about the importance of advocating for her child, the power of online support groups and struggle of navigating a new normal.

My name is Kristine Ritchie, I am from the Central Interior of BC in West Kelowna. I am the parent of an idiopathic pulmonary hypertension child who is 10 years old, and he was diagnosed at four and a half years old on December 12th of 2012.

Brendan has always been a small child and we thought that was pretty normal. One day we decided to go for a walk in November, just up the road from our house. It wasn't a strenuous walk and it was probably about 10 o'clock in the morning. We walked across the street and probably about 500 meters up the path. Brendan said to his dad, "Daddy I'm tired." He let out a whimper and then he fell to the ground. He basically had a seizure. His eyes rolled back in his head, he couldn't talk, he didn't really know where he was. That started our journey.

We took him to our local hospital, Kelowna General Hospital and we waited for the pediatrician on call, because he wanted to come check him out. About two, two and a half weeks later, we had an echocardiogram and we got the results that he had pulmonary hypertension. We were flown down to Vancouver two days later and we stayed at BC Children's for nine days with a myriad of tests and now we actually travel from Kelowna to Edmonton to his PH specialist.

With the medication, they knew what they wanted to start him on. He started on sildenafil and because he was only four and a half years old, he didn't know how to swallow any pills. We were able to compound it. However, our insurance companies would not cover the medication due to the type of medication that it was. Our insurance company wouldn't cover erectile dysfunction drugs, so they refused the medication for him. It took three months basically of full-time letter writing and advocacy and fighting to get the medication covered from our insurance.

Basically, I was reaching out to anyone. I wanted to do anything I could for my child. When he was first diagnosed in Kelowna the pediatrician said, "Don't google pulmonary hypertension." The first thing I did that night after all the lights had gone off was google pulmonary hypertension. I figured out what it was and I just sat in the hospital room just not knowing what to do.

When we went to Vancouver, we had a great medical team and I'll [always] remember Dr. Sanatani. Although we don't get followed by him anymore, I will always remember he said that if I didn't advocate for my son, no one else would and I've remember that phrase for the last almost six years.

So I wrote letters to insurance, I wrote letters to my human resources. In the past six years I've had write letters throughout a myriad of circumstances to various levels within our school, our school district, our school board, the Minister of Education for BC. Just for help on so many different things that it has at some points been more than a full-time job advocating for my child.

My saving grace basically is a Facebook group: (Families of children with Pulmonary Hypertension). In the Central Okanagan, the Interior, there aren't hardly any other families that have children with pulmonary hypertension. Social media has been a great resource for me. I know not everyone is into social media, but the amount of people that you can connect with and everyone has a story, and everyone has been so uplifting and always there. Because it's all over the world, if you ask a question in the middle of the night, someone is going to be awake somewhere to help you answer your question and be a support for you. I feel like social media has been second to none for a caregiver such as myself.

Then also trying to get involved to your comfort level. Going and meeting other people. Reaching out to not only meet them, but listening to other people's stories and learning from other people, because so many people have traveled this road before us. If you can ask questions and follow the advice of others and the stories and the situations that others have been through, I think that it empowers you as a parent. If you're a patient and you find the patient support groups, then that I can empower you as a patient as well.

A lot of people say that it's creating a new normal and at the beginning there's so much going on, but just knowing that a new normal will eventually come down the road, sometimes it might take a few months. Right now, we are adjusting to the life of a subcutaneous pump, so still learning how to see my son attached to tubing and attached to a pump is still not quite normal yet. I know it's going to get there, but that's my biggest thing is a new normal will come, just give yourself time to adjust to everything that's going on.

Kids are incredibly resilient and resourceful. My son has barely complained even after a site change when sometimes he can't feed himself or do up his seat belt or he needs help getting dressed. He doesn't complain. We did have to work on him getting wet and into a pool, so just watching him be so proud of himself and take ownership and helping him advocate for himself. I think we can learn a lot from kids just watching them and how happy they are and how adaptive they are to everything.

Probably the only other thing that I would add, if I can do a broad spectrum… I think for kids, (because of course I have a pediatric PH patient), getting kids to meet other kids like themselves. Going to the Painted Turtle Camp if you can, talking to other people in Clinic Day because a lot of them are there for the same reason you are. Brendan thrives on meeting other kids and knowing their names and what have they got and it really helped him being able to see other children on a pump before him or seeing what they can do. Now he can share that he still goes tubing behind a boat, he goes golfing, he plays baseball and all of this is with an IV in his arm and a pump around his stomach.

]]>Pediatric PH caregiver Kristine Ritchie discusses her son Brendan’s pulmonary hypertension diagnosis. Kristine talks about the importance of advocating for her child, the power of online support groups and struggle of navigating a new normal.

Diagnosed with IPAH almost 16 years ago, Canadian Sonya Collins discusses spending about four years questioning her shortness of breath (SOB) and severe fluid retention. Sonya details why she became a PHA Canada Ambassador and how her short and long term goals have shifted over the years thanks to her PH therapies.

My name is Sonya Collins, and I'm from Paradise Newfoundland, Canada. I am a PH patient. I have been diagnosed for almost 16 years and have recently become an ambassador for PHA Canada, where I'm here to fight for those who can't fight for themselves.

I'm pretty sure that I had PH at least four years prior to being diagnosed. Upon diagnosis, when everything was put in my face of how these are the symptoms. This is what was going on. I was textbook. It started with fluid buildup, and I was pregnant. But, when you're pregnant you have fluid retention. So, then I guess after I had my child, my first, I started to feel a little bit better.

Then I got pregnant again, and on my second child the breathing was much more difficult. Pregnancy induced asthma is what they call that. But, that didn't help. Then after I had my second child, I went to my GP and she said, “you know you're tired. You're not well and everything. You have two babies.” So, it was passed off as just being a tired new mom. My youngest daughter was 20 months old when I got diagnosed.

I was at a conference. I used to be a travel agent and teach travel and tourism, and I saw that all these people were walking past me. People who were much older and maybe more unhealthy than I was. And, I thought this is not right. I'm stopping and looking at pictures to catch my breath so no one sees that I'm out of breath. And, I'm embarrassed, and of course I had gained weight. But a lot of that weight was fluid. So, then it was like, you know what? There's really something wrong here.

That's when I went to my GP, and I was like, “I'm coughing even just talking in a normal conversation, just brushing my teeth, I'm out of breath. I'm hanging over the sink.” She said, “No. This is not normal.” After a month, I got into emergency testing, so an MRI, CT scan and they literally walked me from the scan to the emergency. Through three days of testing told me that I was Stage 4A, and my pressures were around 120 and that I needed a double lung and a heart transplant, but I was too unwell to survive the travel, the transplant, everything. So, that I had about six to twelve months to live.

I would pray, just let me see my 20 month old turn two years old. And then I would pray, just let me see my little four year old go to school, first day of kindergarten. So, those were my short term and my long term goals. Then after about a year, as I started to feel better, I realized, no. They were the wrong goals. My short term goal is to see my children graduate from high school, and my long term goal is be a grandparent. So, my short term goal is about to come true in June when my youngest graduates from high school. And now I have to change my goals again. So, now my new short term goal is to be a grandmother, and my long term goal is to be a great-grandmother.

My cardiologist actually wrote a letter to the government saying that I needed financial assistance to care for children in daycare because I was too unwell to take care of them. So, they did do that, and I would sit at home all day long two minutes away from where my children were in daycare and feel guilty, because I had sent them to daycare and here I am just sitting down all day long.

But, then they would come home at suppertime, dinnertime, and I would think, wow. That's why they're there, because just getting them dinner is making me tired and out of breath and now I have to bathe them and I have to get them ready for bed. So, that was the reason why. But, I still went through a lot of guilt about that. When I would see kids walking down the street, maybe the parents were pulling the children in their slides. I would look out and think, I wish I could do that. I can't do that, and I'd feel guilt.

My oldest daughter just went to University, and she wrote me a letter before she left. She said, I looked as I couldn't do those things. And that my mom was sick in bed. But, I thought okay, I'm sick in bed, and I can't be out doing those things with my children. She looked at is as I had snuggle time with my mom. So, I guess that you can take a positive out of everything. I hope with my children that if I taught them anything is to be positive and I see the world so much better now. And, that there's a good reason for everything, and not that everything happens for a reason. Because I don't really like that saying. But, I think that you can take something good out of everything that happens.

When I started oral therapy, a few days after I was diagnosed in 2003. They said, this is a new drug that was just approved in Newfoundland, and there's no long term information on it [or] data on it. So, we just have to hope for the best when they give this to you now. Fortunately, I did get the best out of it, and that lasted for me for about eight years. Then they had to add a secondary. So, dual therapy. That didn't do so great with me. So, then it was decided just over four years ago that I needed IV therapy.

So, I went on the IV therapy. Then a couple years ago, about two years ago, I heard about a new oral therapy that was similar to the IV, just a different deliverance. And, I lobbied to try and get that for myself. We did, and I just spent a couple weeks in ICU about a month ago. And, I'm now IV free, pump free, and I'm just taking oral therapy. So far I'm doing great.

I guess initially it started with me wanting to go on the oral therapy. I started to fight for it. Then it became more about, okay this is great. I'm fighting for me, but there has to be so many other people out there who don't know how to fight or don't even know that they can fight. Because I was diagnosed for so long, and I was diagnosed before PHA Canada was around or social media. I was used dealing with this disease on my own and battling for myself.

So, then I realized that I don't need to be an Ambassador or be a part of PHA Canada for me. I needed to be a PH ambassador for everybody else. Right now I guess my short term goals are to reach out to people maybe even like me who don't thing that they need us. Whether that's just because of denial or they just don't think that they need us, to reach out to them and show them how we can be of support to them, if not necessarily a patient, but a caregiver.

In my opinion, the caregivers are the heroes, because I think it's harder to watch someone else you love go through something than it is to deal with it yourself. So, maybe it's just going to be for the caregivers over patient even.

My long term goal is that nobody should have to fight for their drugs. Nobody should have to struggle financially. That would be probably my long term goal.

Always have hope, because without hope you have nothing. Stay positive, and reach out to people. There's more people out there. There's more good out there than there's bad, and there's more people out there to help than you realize. Even though friends and family don't exactly understand what you're going through, we understand what you're going through or can empathize with what you're going through.

Diagnosed with IPAH almost 16 years ago, Canadian Sonya Collins discusses spending about four years questioning her shortness of breath (SOB) and severe fluid retention. Sonya details why she became a PHA Canada Ambassador and how her short and long term goals have shifted over the years thanks to her PH therapies.

My name is Sonya Collins, and I'm from Paradise Newfoundland, Canada. I am a PH patient. I have been diagnosed for almost 16 years and have recently become an ambassador for PHA Canada, where I'm here to fight for those who can't fight for themselves.

I'm pretty sure that I had PH at least four years prior to being diagnosed. Upon diagnosis, when everything was put in my face of how these are the symptoms. This is what was going on. I was textbook. It started with fluid buildup, and I was pregnant. But, when you're pregnant you have fluid retention. So, then I guess after I had my child, my first, I started to feel a little bit better.

Then I got pregnant again, and on my second child the breathing was much more difficult. Pregnancy induced asthma is what they call that. But, that didn't help. Then after I had my second child, I went to my GP and she said, “you know you're tired. You're not well and everything. You have two babies.” So, it was passed off as just being a tired new mom. My youngest daughter was 20 months old when I got diagnosed.

I was at a conference. I used to be a travel agent and teach travel and tourism, and I saw that all these people were walking past me. People who were much older and maybe more unhealthy than I was. And, I thought this is not right. I'm stopping and looking at pictures to catch my breath so no one sees that I'm out of breath. And, I'm embarrassed, and of course I had gained weight. But a lot of that weight was fluid. So, then it was like, you know what? There's really something wrong here.

That's when I went to my GP, and I was like, “I'm coughing even just talking in a normal conversation, just brushing my teeth, I'm out of breath. I'm hanging over the sink.” She said, “No. This is not normal.” After a month, I got into emergency testing, so an MRI, CT scan and they literally walked me from the scan to the emergency. Through three days of testing told me that I was Stage 4A, and my pressures were around 120 and that I needed a double lung and a heart transplant, but I was too unwell to survive the travel, the transplant, everything. So, that I had about six to twelve months to live.

I would pray, just let me see my 20 month old turn two years old. And then I would pray, just let me see my little four year old go to school, first day of kindergarten. So, those were my short term and my long term goals. Then after about a year, as I started to feel better, I realized, no. They were the wrong goals. My short term goal is to see my children graduate from high school, and my long term goal is be a grandparent. So, my short term goal is about to come true in June when my youngest graduates from high school. And now I have to change my goals again. So, now my new short term goal is to be a grandmother, and my long term goal is to be a great-grandmother.

My cardiologist actually wrote a letter to the government saying that I needed financial assistance to care for children in daycare because I was too unwell to take care of them. So, they did do that, and I would sit at home all day long two minutes away from where my children were in daycare and feel guilty, because I had sent them to daycare and here I am just sitting down all day long.

But, then they would come home at suppertime, dinnertime, and I would think, wow. That's why they're there, because just getting them dinner is making me tired and out of breath and now I have to bathe them and I have to get them ready for bed. So, that was the reason why. But, I still went through a lot of guilt about that. When I would see kids walking down the street, maybe the parents were pulling the children in their slides. I would look out and think, I wish I could do that. I can't do that, and I'd feel guilt.

My oldest daughter just went to University, and she wrote me a letter before she left. She said, I looked as I couldn't do those things. And that my mom was sick in bed. But, I thought okay, I'm sick in bed, and I can't be out doing those things with my children. She looked at is as I had snuggle time with my mom. So, I guess that you can take a positive out of everything. I hope with my children that if I taught them anything is to be positive and I see the world so much better now. And, that there's a good reason for everything, and not that everything happens for a reason. Because I don't really like that saying. But, I think that you can take something good out of everything that happens.

When I started oral therapy, a few days after I was diagnosed in 2003. They said, this is a new drug that was just approved in Newfoundland, and there's no long term information on it [or] data on it. So, we just have to hope for the best when they give this to you now. Fortunately, I did get the best out of it, and that lasted for me for about eight years. Then they had to add a secondary. So, dual therapy. That didn't do so great with me. So, then it was decided just over four years ago that I needed IV therapy.

So, I went on the IV therapy. Then a couple years ago, about two years ago, I heard about a new oral therapy that was similar to the IV, just a different deliverance. And, I lobbied to try and get that for myself. We did, and I just spent a couple weeks in ICU about a month ago. And, I'm now IV free, pump free, and I'm just taking oral therapy. So far I'm doing great.

I guess initially it started with me wanting to go on the oral therapy. I started to fight for it. Then it became more about, okay this is great. I'm fighting for me, but there has to be so many other people out there who don't know how to fight or don't even know that they can fight. Because I was diagnosed for so long, and I was diagnosed before PHA Canada was around or social media. I was used dealing with this disease on my own and battling for myself.

So, then I realized that I don't need to be an Ambassador or be a part of PHA Canada for me. I needed to be a PH ambassador for everybody else. Right now I guess my short term goals are to reach out to people maybe even like me who don't thing that they need us. Whether that's just because of denial or they just don't think that they need us, to reach out to them and show them how we can be of support to them, if not necessarily a patient, but a caregiver.

In my opinion, the caregivers are the heroes, because I think it's harder to watch someone else you love go through something than it is to deal with it yourself. So, maybe it's just going to be for the caregivers over patient even.

My long term goal is that nobody should have to fight for their drugs. Nobody should have to struggle financially. That would be probably my long term goal.

Always have hope, because without hope you have nothing. Stay positive, and reach out to people. There's more people out there. There's more good out there than there's bad, and there's more people out there to help than you realize. Even though friends and family don't exactly understand what you're going through, we understand what you're going through or can empathize with what you're going through.

]]>fullEpisode 209 - Sonya CollinsSonya Collins - Pulmonary HypertensionThu, 22 Nov 2018 13:00:00 +0000Diagnosed with IPAH almost 16 years ago, Canadian Sonya Collins discusses spending about four years questioning her shortness of breath (SOB) and severe fluid retention. Sonya details why she became a PHA Canada Ambassador and how her short and long term goals have shifted over the years thanks to her PH therapies.

]]>Diagnosed with IPAH almost 16 years ago, Canadian Sonya Collins discusses spending about four years questioning her shortness of breath (SOB) and severe fluid retention. Sonya details why she became a PHA Canada Ambassador and how her short and long term goals have shifted over the years thanks to her PH therapies.

Pediatric PH caregiver Shannon Reitor discusses her son Adam’s pulmonary hypertension diagnosis. Shannon details lobbying the Canadian government, the challenge of navigating side-effects of unapproved treatments for children with PH and how Adam became the first child in Canada to go on IV therapy.

My name is Shannon Reitor. I am from Edmonton, Alberta, in Canada, and my connection to pulmonary hypertension is through my son, Adam. He was diagnosed the day before his second birthday.

I always knew something was wrong with him. I did the typical parent fight for years, where you go in and you know something [is wrong]. They're not growing, they're not thriving the way they should be. Eventually, he wound up getting bronchial pneumonia, and that led to a hospital visit where they heard a murmur, and that's actually the direction that we wound up going in. So bronchial pneumonia was no longer a concern. Cardiology now was. It got brought in, and the day before his second birthday was his echo cardiogram, so it was his unofficial diagnosis. Two weeks later, was his heart catheterization where he received his official diagnosis.

I remember after that echocardiogram appointment, the nurses actually came to me and asked me if I was okay to drive home and I was like, "Oh, yeah, I'm fine, I'm just going to go have a glass [of wine]," and she looked at me and she said, "With the news you just had, you deserve a bottle." That's when I realized that I'd better find out a bit more about the direction we were about to go in.

The first thing that wound up happening was we obviously had to start putting him onto medications. So now you're dealing with medications, side effects. Unfortunately for him, one of them - bosentan, was not a positive one. He had every severe side effect you could get, which is not common in kids, so we had to figure out a way to switch that under Canadian laws. There weren't really any drugs that were available, so we had to do some special circumstances just to get ambrisentan in. It wasn't even available yet.

Usually, what we have to do is we have to lobby to the government. Through the government, if they give us approval, in order to bring it in, that means that they're going to financially cover it, because it's not under any of the pharmacy guidelines with how much it costs. Then we have to get the actual pharmaceutical company to approve that they're even going to send it, because now they're sending it to a doctor that now has to be responsible for it that is not under their guidelines and their care that have already been approved.

Once we get through that - and it takes time, they wind up sending it out to us. Most of the time it's under trials, so that way we don't wind up having to have it come out of pocket. Then they can use the data that they get from it towards their studies.

He was on subcutaneous Remodulin (treprostinil) for a couple of years. The sites then started to fall apart. He was within the first three kids in Canada to ever go on it. I know it wasn't popular in the United States yet, so really, we didn't know what we were doing or what long-term effects were going to be. His skin didn't hold up and it rejected all the sites. We were lucky if we got three days, so he became the first kid in Canada to go on IV Remodulin (treprostinil), which was a game-changer. It changed how well he felt. He wasn't in pain all the time, so we've experienced both sides of how that goes.

Unfortunately, his skin eventually didn't hold those lines, so we took a risk and he went pump-free for a while, while we fought for selexipag, that had been FDA-approved. It had been Health Canada-approved, but it wasn't available for us to get it, again, due to the politics behind it all.

I had to actually send in all the pictures of all of his gross sites and all of his line infections and everything, and it went to head office, and they sent it to us on them. They didn't even want the government involved by that point in time. They realized that some kids, they just are better candidates for it to try it out, because it wasn't a matter of there were other drugs available. There was nothing else available.

It's usually pretty frightening in the sense that we don't have any precedent set. We don't know what we're doing, we're just winging it and hoping that it works. In that sense, it's terrifying, but then once all the kids afterward start following and they start doing really well, then it feels good to know that him starting on something showed information, at least enough of it that they felt confident to have more kids get that kind of help.

Don't give up hope, because even eight years ago it was a lot harder than it is today, and if we can come this far within the last eight years, I can only imagine what the next couple are going to look like. It's not going to take long before our kids are running around just like anybody else.

Pediatric PH caregiver Shannon Reitor discusses her son Adam’s pulmonary hypertension diagnosis. Shannon details lobbying the Canadian government, the challenge of navigating side-effects of unapproved treatments for children with PH and how Adam became the first child in Canada to go on IV therapy.

My name is Shannon Reitor. I am from Edmonton, Alberta, in Canada, and my connection to pulmonary hypertension is through my son, Adam. He was diagnosed the day before his second birthday.

I always knew something was wrong with him. I did the typical parent fight for years, where you go in and you know something [is wrong]. They're not growing, they're not thriving the way they should be. Eventually, he wound up getting bronchial pneumonia, and that led to a hospital visit where they heard a murmur, and that's actually the direction that we wound up going in. So bronchial pneumonia was no longer a concern. Cardiology now was. It got brought in, and the day before his second birthday was his echo cardiogram, so it was his unofficial diagnosis. Two weeks later, was his heart catheterization where he received his official diagnosis.

I remember after that echocardiogram appointment, the nurses actually came to me and asked me if I was okay to drive home and I was like, "Oh, yeah, I'm fine, I'm just going to go have a glass [of wine]," and she looked at me and she said, "With the news you just had, you deserve a bottle." That's when I realized that I'd better find out a bit more about the direction we were about to go in.

The first thing that wound up happening was we obviously had to start putting him onto medications. So now you're dealing with medications, side effects. Unfortunately for him, one of them - bosentan, was not a positive one. He had every severe side effect you could get, which is not common in kids, so we had to figure out a way to switch that under Canadian laws. There weren't really any drugs that were available, so we had to do some special circumstances just to get ambrisentan in. It wasn't even available yet.

Usually, what we have to do is we have to lobby to the government. Through the government, if they give us approval, in order to bring it in, that means that they're going to financially cover it, because it's not under any of the pharmacy guidelines with how much it costs. Then we have to get the actual pharmaceutical company to approve that they're even going to send it, because now they're sending it to a doctor that now has to be responsible for it that is not under their guidelines and their care that have already been approved.

Once we get through that - and it takes time, they wind up sending it out to us. Most of the time it's under trials, so that way we don't wind up having to have it come out of pocket. Then they can use the data that they get from it towards their studies.

He was on subcutaneous Remodulin (treprostinil) for a couple of years. The sites then started to fall apart. He was within the first three kids in Canada to ever go on it. I know it wasn't popular in the United States yet, so really, we didn't know what we were doing or what long-term effects were going to be. His skin didn't hold up and it rejected all the sites. We were lucky if we got three days, so he became the first kid in Canada to go on IV Remodulin (treprostinil), which was a game-changer. It changed how well he felt. He wasn't in pain all the time, so we've experienced both sides of how that goes.

Unfortunately, his skin eventually didn't hold those lines, so we took a risk and he went pump-free for a while, while we fought for selexipag, that had been FDA-approved. It had been Health Canada-approved, but it wasn't available for us to get it, again, due to the politics behind it all.

I had to actually send in all the pictures of all of his gross sites and all of his line infections and everything, and it went to head office, and they sent it to us on them. They didn't even want the government involved by that point in time. They realized that some kids, they just are better candidates for it to try it out, because it wasn't a matter of there were other drugs available. There was nothing else available.

It's usually pretty frightening in the sense that we don't have any precedent set. We don't know what we're doing, we're just winging it and hoping that it works. In that sense, it's terrifying, but then once all the kids afterward start following and they start doing really well, then it feels good to know that him starting on something showed information, at least enough of it that they felt confident to have more kids get that kind of help.

Don't give up hope, because even eight years ago it was a lot harder than it is today, and if we can come this far within the last eight years, I can only imagine what the next couple are going to look like. It's not going to take long before our kids are running around just like anybody else.

]]>Pediatric PH caregiver Shannon Reitor discusses her son Adam’s pulmonary hypertension diagnosis. Shannon details lobbying the Canadian government, the challenge of navigating side-effects of unapproved treatments for children with PH and how Adam became the first child in Canada to go on IV therapy.

Angela Michelle is a professional photographer and yoga instructor living in San Antonio, Texas. She was diagnosed with CTEPH in January 2018 after years of experiencing symptoms. Angela underwent PTE surgery in August of 2018 at UCSD.

My name is Angela Michelle. I am from San Antonio, Texas, and I was diagnosed with CTEPH, which is a form of pulmonary hypertension.

How I was diagnosed is actually quite a story. I began getting short of breath probably about 2013, and I kept bringing it up to my doctors. I was told it was allergies, it was asthma, respiratory infections, it was just that I was overweight. I would take whatever meds they gave me. I'd use inhalers. Nothing worked. I would try to work out. I was developing an intolerance to exercise, and so this continued on for quite a few years.

Then in 2016, I actually had a stroke. At that time, they realized I had multiple pulmonary embolisms, as well as my carotids were blocked. They decided to try to figure out what would make a then 38-year-old woman just throw all these clots, so it was determined I have antiphospholipid syndrome, and that was the first I'd ever heard of this particular disease. That was 2016, and I was short of breath at the time, and they told me, oh, it'll get better. They'll dissolve on their own, and so I left the hospital, continued on with my life. I was really lucky in that my stroke didn't leave me with a whole lot of physical limitations. It did leave me completely blind in one eye, but I found I was able to adapt to that.

So as I continued on, I just kept getting more and more short of breath. I am a yoga instructor, and so I would notice that even during yoga class, I was way out of breath more so than usual. I again brought it up to my doctors and they decided to send me to a pulmonologist, but it was going to be a long wait, and in the meantime, I ended up developing a cyst that needed to be removed, and it was totally unrelated to anything, but because of me being on blood thinners, it did require a hospitalization.

While I was there, and I was going to get put under to remove the cyst, they realized that my O2s were really low, and it was so low that they decided not to put me completely under, so I had to have the operation awake. Then at that time, they decided to send me home on oxygen, and when I kept trying to figure out like, well, why is this happening and what it is, they didn't seem to know, and they're like, "you'll just have to wait to see the pulmonologist." This happened a little over a year after my stroke, so this was July of 2017.

While I was in the hospital, I was very active on social media, Facebook and Instagram, and I shared a lot of my story, especially my health condition because I actually had two hospitals misdiagnose my stroke, and it was a third hospital that caught it. so I feel like there's a lot of misconceptions about that as well. So I am very active in telling my story. At that time, I was writing on Facebook and I was sharing that I was in the hospital and my low O2s, and it turns out an old friend of mine who I actually knew from middle school and high school had become a heart surgeon, and he was actually working out in L.A. He privately messaged me and suggested that they look into pulmonary hypertension, particularly CTEPH. He explained that it was really rare, but that I kind of fit the bill for it considering my antiphospholipid blood clotting disorder, that I was out of breath with no known cause, and so I was the one to originally bring it up to my doctors here in San Antonio.

At this time, I was already on oxygen 24/7, and the disease had progressed to a point that I couldn't even walk across the room without getting really short of breath, feeling like I was going to pass out and my heart rate skyrocketing. When I brought it up to my doctors locally, they were hesitant because they were like, "well, it's really rare", and they're like, "well, maybe, but let's run tests." I went through the whole battery of tests, and it wasn't until I had my right heart catheterization in January of 2018, and at the time the cardiologist that performed it, she said I didn't have pulmonary hypertension. So I was kind of like, well, that's good, but now we're back at square one.

Well, then my follow-up appointment of the pulmonologist, he was like, "no, you do have it, and I'm sending you to a PH specialist." So I ended up at a PH specialist here in San Antonio, and as soon as I met with him, he asked me what I knew about the disease, and at this point because my friend had told me about it, I had months of looking it up online and learning about it, so I told him what I knew, and he said I was pretty well informed and that he was sending me as a recommendation to have PTE surgery in San Diego.

I did have PTE surgery on August 7, 2018, so I'm about 10, 11 weeks out of surgery. I'm doing much better. I do still use oxygen on exertion. I'm currently in pulmonary rehabilitation, and I wear it when I work out, but I'm not needing it on my day-to-day activities and I have seen a huge difference.

After my stroke was misdiagnosed, I realized I had to take my health into my own hands. Prior to that, I relied on what the doctors said, I felt like they knew best, I didn't have a medical degree, and after two hospitals had misdiagnosed me, I knew something was wrong, but I didn't listen to my gut. I listened to the doctors. After that happened, I realized that it was really important that I be my own health advocate.

When my friend suggested that this was a disease I could have, I decided to go full force and use my energy to understand this disease, to figure out if it's what I had, and to really push my doctors. I really do feel like in a lot of ways I had to advocate for the tests that I needed to have done. I was lucky to have a friend who knew about this disease. He knew what tests I needed to have done to get the diagnosis. He was a text away from answering any of my questions, and he told me what tests to ask for, what questions to ask for, and I was able to send him copies of my test results and he could help me decipher them. I was really lucky to have that, and not everyone has that access to someone who can help guide them.

I just think that there's a lot of misinformation and misconceptions, and I don't think a lot of doctors understand this disease yet. Just like my cardiologist, I don't even know if she knows what it is, because according to her, I didn't have it, and I obviously did and do, so I just think there's a lot of information that is still not being put out and getting to the doctors so that they diagnose people properly.

Life has definitely opened up to me. It's amazing how I feel, I'm not short of breath all the time. I can do things like walk my dog or go to the grocery store and not be tied to an oxygen machine. For the past year and a half I was on oxygen 24/7, so I don't think you realize until you've gone through this, the amount of freedom that you had taken from you and then that you get back after, so I feel very free and very lucky to be on this side. I was really scared of the surgery because I do have a blood clotting disorder and I knew the risks going into that, so just to be on the other side, the anxiety and the I guess frustration of it all has really dissipated.

Advocacy is really important to me right now because it did take so long to get diagnosed, even though it was about I guess two years/a year and a half from thrombosis, at least known thrombosis to the CTEPH. I had been experiencing symptoms five years before that. Because of my missed stroke, I am really big on advocacy and educating the community, knowing the signs of different diseases, understanding our body, listening to our body, and learning how to be our own health advocate.

I would love to help patients learn how to navigate their health and the system, and so I am working on material to actually teach people how to do that, how to take the right notes and where to research and how to research, and how to reach out and how to talk to your doctors. I feel like that's really important, and we need to be responsible for our own health and not just hand it all over to someone who sees us for five/ten minutes every once in a while.

Also understand that for those of us living with these diseases, we live a very different life. A little bit of empathy, compassion can go a long way to help us in our world and navigating what we go through. A little bit of self-love and compassion for ourselves too that this is hard hands that we have been dealt, but that ultimately the choice is ours in how we choose to live our life and how we choose to deal with what we've been dealt. I always say it is what we make it in life, and so I feel like me going through this was part of me experiencing what I had to to lead me to where I'm at, and where I'm at is really wanting to help others now.

Angela Michelle is a professional photographer and yoga instructor living in San Antonio, Texas. She was diagnosed with CTEPH in January 2018 after years of experiencing symptoms. Angela underwent PTE surgery in August of 2018 at UCSD.

My name is Angela Michelle. I am from San Antonio, Texas, and I was diagnosed with CTEPH, which is a form of pulmonary hypertension.

How I was diagnosed is actually quite a story. I began getting short of breath probably about 2013, and I kept bringing it up to my doctors. I was told it was allergies, it was asthma, respiratory infections, it was just that I was overweight. I would take whatever meds they gave me. I'd use inhalers. Nothing worked. I would try to work out. I was developing an intolerance to exercise, and so this continued on for quite a few years.

Then in 2016, I actually had a stroke. At that time, they realized I had multiple pulmonary embolisms, as well as my carotids were blocked. They decided to try to figure out what would make a then 38-year-old woman just throw all these clots, so it was determined I have antiphospholipid syndrome, and that was the first I'd ever heard of this particular disease. That was 2016, and I was short of breath at the time, and they told me, oh, it'll get better. They'll dissolve on their own, and so I left the hospital, continued on with my life. I was really lucky in that my stroke didn't leave me with a whole lot of physical limitations. It did leave me completely blind in one eye, but I found I was able to adapt to that.

So as I continued on, I just kept getting more and more short of breath. I am a yoga instructor, and so I would notice that even during yoga class, I was way out of breath more so than usual. I again brought it up to my doctors and they decided to send me to a pulmonologist, but it was going to be a long wait, and in the meantime, I ended up developing a cyst that needed to be removed, and it was totally unrelated to anything, but because of me being on blood thinners, it did require a hospitalization.

While I was there, and I was going to get put under to remove the cyst, they realized that my O2s were really low, and it was so low that they decided not to put me completely under, so I had to have the operation awake. Then at that time, they decided to send me home on oxygen, and when I kept trying to figure out like, well, why is this happening and what it is, they didn't seem to know, and they're like, "you'll just have to wait to see the pulmonologist." This happened a little over a year after my stroke, so this was July of 2017.

While I was in the hospital, I was very active on social media, Facebook and Instagram, and I shared a lot of my story, especially my health condition because I actually had two hospitals misdiagnose my stroke, and it was a third hospital that caught it. so I feel like there's a lot of misconceptions about that as well. So I am very active in telling my story. At that time, I was writing on Facebook and I was sharing that I was in the hospital and my low O2s, and it turns out an old friend of mine who I actually knew from middle school and high school had become a heart surgeon, and he was actually working out in L.A. He privately messaged me and suggested that they look into pulmonary hypertension, particularly CTEPH. He explained that it was really rare, but that I kind of fit the bill for it considering my antiphospholipid blood clotting disorder, that I was out of breath with no known cause, and so I was the one to originally bring it up to my doctors here in San Antonio.

At this time, I was already on oxygen 24/7, and the disease had progressed to a point that I couldn't even walk across the room without getting really short of breath, feeling like I was going to pass out and my heart rate skyrocketing. When I brought it up to my doctors locally, they were hesitant because they were like, "well, it's really rare", and they're like, "well, maybe, but let's run tests." I went through the whole battery of tests, and it wasn't until I had my right heart catheterization in January of 2018, and at the time the cardiologist that performed it, she said I didn't have pulmonary hypertension. So I was kind of like, well, that's good, but now we're back at square one.

Well, then my follow-up appointment of the pulmonologist, he was like, "no, you do have it, and I'm sending you to a PH specialist." So I ended up at a PH specialist here in San Antonio, and as soon as I met with him, he asked me what I knew about the disease, and at this point because my friend had told me about it, I had months of looking it up online and learning about it, so I told him what I knew, and he said I was pretty well informed and that he was sending me as a recommendation to have PTE surgery in San Diego.

I did have PTE surgery on August 7, 2018, so I'm about 10, 11 weeks out of surgery. I'm doing much better. I do still use oxygen on exertion. I'm currently in pulmonary rehabilitation, and I wear it when I work out, but I'm not needing it on my day-to-day activities and I have seen a huge difference.

After my stroke was misdiagnosed, I realized I had to take my health into my own hands. Prior to that, I relied on what the doctors said, I felt like they knew best, I didn't have a medical degree, and after two hospitals had misdiagnosed me, I knew something was wrong, but I didn't listen to my gut. I listened to the doctors. After that happened, I realized that it was really important that I be my own health advocate.

When my friend suggested that this was a disease I could have, I decided to go full force and use my energy to understand this disease, to figure out if it's what I had, and to really push my doctors. I really do feel like in a lot of ways I had to advocate for the tests that I needed to have done. I was lucky to have a friend who knew about this disease. He knew what tests I needed to have done to get the diagnosis. He was a text away from answering any of my questions, and he told me what tests to ask for, what questions to ask for, and I was able to send him copies of my test results and he could help me decipher them. I was really lucky to have that, and not everyone has that access to someone who can help guide them.

I just think that there's a lot of misinformation and misconceptions, and I don't think a lot of doctors understand this disease yet. Just like my cardiologist, I don't even know if she knows what it is, because according to her, I didn't have it, and I obviously did and do, so I just think there's a lot of information that is still not being put out and getting to the doctors so that they diagnose people properly.

Life has definitely opened up to me. It's amazing how I feel, I'm not short of breath all the time. I can do things like walk my dog or go to the grocery store and not be tied to an oxygen machine. For the past year and a half I was on oxygen 24/7, so I don't think you realize until you've gone through this, the amount of freedom that you had taken from you and then that you get back after, so I feel very free and very lucky to be on this side. I was really scared of the surgery because I do have a blood clotting disorder and I knew the risks going into that, so just to be on the other side, the anxiety and the I guess frustration of it all has really dissipated.

Advocacy is really important to me right now because it did take so long to get diagnosed, even though it was about I guess two years/a year and a half from thrombosis, at least known thrombosis to the CTEPH. I had been experiencing symptoms five years before that. Because of my missed stroke, I am really big on advocacy and educating the community, knowing the signs of different diseases, understanding our body, listening to our body, and learning how to be our own health advocate.

I would love to help patients learn how to navigate their health and the system, and so I am working on material to actually teach people how to do that, how to take the right notes and where to research and how to research, and how to reach out and how to talk to your doctors. I feel like that's really important, and we need to be responsible for our own health and not just hand it all over to someone who sees us for five/ten minutes every once in a while.

Also understand that for those of us living with these diseases, we live a very different life. A little bit of empathy, compassion can go a long way to help us in our world and navigating what we go through. A little bit of self-love and compassion for ourselves too that this is hard hands that we have been dealt, but that ultimately the choice is ours in how we choose to live our life and how we choose to deal with what we've been dealt. I always say it is what we make it in life, and so I feel like me going through this was part of me experiencing what I had to to lead me to where I'm at, and where I'm at is really wanting to help others now.

]]>fullEpisode 207 - Angela MichelleAngela Michelle - CTEPHThu, 15 Nov 2018 13:00:00 +0000Angela Michelle is a professional photographer and yoga instructor living in San Antonio, Texas. She was diagnosed with CTEPH in January 2018 after years of experiencing symptoms. Angela underwent PTE surgery in August of 2018 at UCSD.

]]>Angela Michelle is a professional photographer and yoga instructor living in San Antonio, Texas. She was diagnosed with CTEPH in January 2018 after years of experiencing symptoms. Angela underwent PTE surgery in August of 2018 at UCSD.

]]>09:48cleanblood,research,heart,awareness,asthma,breathless,rare,ph,pulmonary,raredisease,clots,catheterization,pulmonaryhypertension,phaware,cteph,lungdiseaseAngela Michelle is a professional photographer and yoga instructor living in San Antonio, Texas. She was diagnosed with CTEPH in January 2018 after years of experiencing symptoms. Angela underwent PTE surgery in August of 2018 at UCSD.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us @phaware #phaware #phawareMD #CTEPH #CTEPHday #ClinicalTrials @CTEPHAdvisor @BayerPharma @BayerUS @antidote_me
*Photo Credit: II Sapphires38fullphaware global associationRobert Frantz, MD - phaware® interview 206Tue, 13 Nov 2018 13:00:00 +0000

Robert P. Frantz is the director of the Mayo Pulmonary Hypertension Clinic, which has a robust referral practice of patients with pulmonary arterial hypertension. Dr. Frantz has served as the Mayo site PI on numerous multicenter trials in PH including the majority of trials resulting in FDA approved PH therapies. He also performs complex hemodynamic studies in the catheterization laboratory including right heart catheterizations for patients with pulmonary arterial hypertension and also patients with left heart failure. He also evaluates and manages patients with CTEPH, with particular interest in the role of balloon pulmonary angioplasty.

My name is Dr. Robert Frantz, and I am the director of the Pulmonary Hypertension Clinic at the Mayo Clinic in Rochester, Minnesota and I've been here over 20 years. I really spend a lot of time taking care of patients with various forms of pulmonary hypertension. One form of pulmonary hypertension that we're seeing more often than we used to is what we call chronic thromboembolic pulmonary hypertension [CTEPH].

That can look just like pulmonary hypertension of other causes in terms of having elevated pulmonary artery pressures and normal left heart filling pressures. Then the development of shortness of breath and failure of the right side of the heart leading to fluid retention and poor activity tolerance. But people don't come waving a flag saying, "Hey, the reason I have pulmonary hypertension is because part of my lung vessels are plugged up with old clots that I didn't know I had." So it is a form of pulmonary hypertension that can be overlooked.

We often say that nobody should be diagnosed with idiopathic pulmonary arterial hypertension (or pulmonary hypertension of unknown cause), without making sure they don't have clots in their lungs. The interesting thing is that many patients could have obstruction of the lung vessels with old fibrotic clots and never have had a clinical history of a clot in their leg veins or having had pulmonary emboli (clots into the lungs) and still have the condition, because it can be silent.

Our lungs are like big filters. So we could have a clot come out of our leg vein during a long airplane ride. It could go flying up to the lungs and plug a lung vessel up. A lot of the times that clot would dissolve, but some of the time, instead of dissolving, it forms a scar that then obstructs that segment of the lung. If enough of those segments get obstructed, finally the cross-sectional area of the lung starts to go down and then it limits flow through the lungs.

The other thing that we know about this condition is that those fibrotic old clots don't dissolve with blood thinners. You can prevent new ones from developing it, but they're not really clots in the sense that you think of in terms of a red thrombus that is mushy and will easily move out of the way or dissolve. The body is clever about these things and goes in there sometimes and tries to heal it up, but actually scars it up and it creates an obstruction. That combined with constriction of the lung vessels may be triggered by the lining of the lung vessels not working quite right, and that's the reason that certain medications that are utilized in chronic thromboembolic pulmonary hypertension actually work, because they can relax the lung vessels to some extent, but they don't take away the obstruction.

Traditionally, the only good option has been to do open surgery under cardiopulmonary bypass and deep circulatory arrest where you cool down the brain and stop circulation and have a surgeon shell the old fibrotic clot out of the lung vessels, which can be a very effective procedure. But it’s obviously quite invasive. Many patients who have this condition may not be candidates for it because the clots are too far out into the periphery of the lung and can't be reached, or they have other conditions that make them not a good operative candidate in terms of risk.

So the other options for that are either a medication, and one specific medication called riociguat is approved to relax the lung vessels to try to help that condition, and other medications that we use for other forms of pulmonary hypertension can be useful too. But that medication only takes us so far in somebody who has inoperable or residual thromboembolic disease after prior surgery.

So balloon pulmonary angioplasty is taking techniques as interventional cardiologists we've learned to use in the coronary circulation; putting wires and balloons across areas of narrowing and inflating them to make things less narrow. It has been adapted to lung circulation, where if you can get a wire across one of these narrowing in the lung vessels, then you can put a balloon across it and restore flow to that segment and thereby improve the pulmonary hypertension and the efficiency of the lungs. That technique at an expert center in the last number of years has been shown to have relatively low risk, although not zero, and really good results. So we've been doing quite a lot of that here at the Mayo Clinic in recent years.

I think it's like any newer technique that starts in a small number of centers that get very familiar with it, and then depending on how readily the techniques can be learned and applied, will then spread to other centers. So the nature of this procedure at the present time is that a relatively small number of centers are doing significant numbers of these procedures in the United States, but many centers that have interventional cardiology programs are highly interested in it and are often coming to visit here to try to learn how to do it or visiting a couple of other centers in the country that do a fair amount of this to try to learn the technique.

I would say that it's evolving with it having been in the United States over a decade ago, but maybe not as readily as with current equipment and not maybe quite as successfully. Then, really, the Japanese picked it up and started doing a lot of cases over Japan and demonstrated that it could be done quite effectively. Now the rest of the world is picking it up. Right now in the United States, there's a handful of centers that are doing substantial numbers of procedures, but there's intense interest in it. So I think it will continue to spread across other centers overtime.

We try to emphasize the idea of an expert multidisciplinary approach, where somebody who is really familiar with the options of open surgery, medical therapy and balloon pulmonary angioplasty is involved in each patient's decision making. So the ideal circumstance would be a person who has this diagnosis of chronic thromboembolic pulmonary hypertension is considered at an expert center with regard to whether they're a candidate for open surgery with regard to whether the vessels that are involved are reachable by a surgeon, and [if] that the person is suitable or not.

If they are not found to be suitable for open surgery, then I think they should really be considered for the balloon procedure, because it has, in my sense, a better opportunity of really taking care of the problem with regard to restoring flow to the obstructive segments as supposed to just relaxing the segments that remain open. So we often end up combining the two things of medical therapy to relax the vessels that are still open, plus balloon work. Then seeing whether we can then takeaway the medications if the balloon work is successful enough to improve the flow to a point where they may not be necessary.

On the other hand, in some patients, if the pulmonary hypertension is not too severe when we start with them, then we might do a balloon work alone and never really need the medications, which is nice in a way, because if you're going to use a lifelong medication, then that's going to have potential for side effects and substantial expense, and if you can avoid that, it's a good thing. So I think every patient should be considered for open surgery, balloon work and medication at a center where those decisions can be made properly. If they're not candidates for open surgery, they should be considered for balloon angioplasty.

Honestly, there are relatively very few of the patients with chronic thromboembolic pulmonary hypertension that we've looked at as potential candidates for balloon angioplasty who felt are not candidates for it. Most of the time, there are vessels that could be crossed and the flow can be improved. If the kidneys are really not in good shape, and especially if they're diabetic, then the risk of the contrast we need in order to visualize the vessels well might not be worth it.

So the kind of patient I would not do in would be a diabetic patient with kidney impairment, where you know if you give them quite a lot of contrast, you put them at risk at putting them onto dialysis or something because of renal failure. This is a procedure where you usually need to do multiple sessions. You can't open everything up once. So it might be several sessions of work each one requiring contrast. So if the kidneys are in trouble, you can really hurt them, and maybe those patients are better off just with medical therapy.

I can't emphasize enough how important a V/Q scan is, which is the nuclear scan where you inhale something that shows where the air is going and you have something injected that shows where the blood is going. Those should match like a hand and a glove, and areas that have ventilation but don't have good flow are areas that may be obstructed with clot.

The critical issue is that we're very good at using CT to look for acute clots in somebody who's in the hospital in the emergency room with shortness of breath and have potential for large clots that are easily seen on CT imaging. But the chronic clots that are often pretty far out are easily missed on a CT scan done with thick sections where the number of cross-sections taken isn't close enough together to not miss the webs and bands and maybe reviewed by a radiologist that isn't very familiar with the appearance of chronic old clots that are peripheral.

I've seen multiple patients who were felt to have idiopathic pulmonary arterial hypertension sometimes have been treated for years, and that was on the basis having at a CT that had not shown clots. Then they've come for a second opinion at some point and I’ve said, "Gosh! They never had a ventilation/perfusion lung scan. Let's do that," and find out lo and behold they have chronic thromboembolic pulmonary hypertension, were felt to have idiopathic pulmonary arterial hypertension, because of this issue of the sensitivity of CT scanning being less for findings of chronic clot than a perfusion lung scan.

So if we do a perfusion lung scan that shows evidence of possible clots, then we do an invasive pulmonary angiography, where we put a catheter out in the pulmonary artery and take pictures directly in order to look at the clots in a more distinct way than we can with either CT or with a nuclear perfusion scan. A very important point.

Robert P. Frantz is the director of the Mayo Pulmonary Hypertension Clinic, which has a robust referral practice of patients with pulmonary arterial hypertension. Dr. Frantz has served as the Mayo site PI on numerous multicenter trials in PH including the majority of trials resulting in FDA approved PH therapies. He also performs complex hemodynamic studies in the catheterization laboratory including right heart catheterizations for patients with pulmonary arterial hypertension and also patients with left heart failure. He also evaluates and manages patients with CTEPH, with particular interest in the role of balloon pulmonary angioplasty.

My name is Dr. Robert Frantz, and I am the director of the Pulmonary Hypertension Clinic at the Mayo Clinic in Rochester, Minnesota and I've been here over 20 years. I really spend a lot of time taking care of patients with various forms of pulmonary hypertension. One form of pulmonary hypertension that we're seeing more often than we used to is what we call chronic thromboembolic pulmonary hypertension [CTEPH].

That can look just like pulmonary hypertension of other causes in terms of having elevated pulmonary artery pressures and normal left heart filling pressures. Then the development of shortness of breath and failure of the right side of the heart leading to fluid retention and poor activity tolerance. But people don't come waving a flag saying, "Hey, the reason I have pulmonary hypertension is because part of my lung vessels are plugged up with old clots that I didn't know I had." So it is a form of pulmonary hypertension that can be overlooked.

We often say that nobody should be diagnosed with idiopathic pulmonary arterial hypertension (or pulmonary hypertension of unknown cause), without making sure they don't have clots in their lungs. The interesting thing is that many patients could have obstruction of the lung vessels with old fibrotic clots and never have had a clinical history of a clot in their leg veins or having had pulmonary emboli (clots into the lungs) and still have the condition, because it can be silent.

Our lungs are like big filters. So we could have a clot come out of our leg vein during a long airplane ride. It could go flying up to the lungs and plug a lung vessel up. A lot of the times that clot would dissolve, but some of the time, instead of dissolving, it forms a scar that then obstructs that segment of the lung. If enough of those segments get obstructed, finally the cross-sectional area of the lung starts to go down and then it limits flow through the lungs.

The other thing that we know about this condition is that those fibrotic old clots don't dissolve with blood thinners. You can prevent new ones from developing it, but they're not really clots in the sense that you think of in terms of a red thrombus that is mushy and will easily move out of the way or dissolve. The body is clever about these things and goes in there sometimes and tries to heal it up, but actually scars it up and it creates an obstruction. That combined with constriction of the lung vessels may be triggered by the lining of the lung vessels not working quite right, and that's the reason that certain medications that are utilized in chronic thromboembolic pulmonary hypertension actually work, because they can relax the lung vessels to some extent, but they don't take away the obstruction.

Traditionally, the only good option has been to do open surgery under cardiopulmonary bypass and deep circulatory arrest where you cool down the brain and stop circulation and have a surgeon shell the old fibrotic clot out of the lung vessels, which can be a very effective procedure. But it’s obviously quite invasive. Many patients who have this condition may not be candidates for it because the clots are too far out into the periphery of the lung and can't be reached, or they have other conditions that make them not a good operative candidate in terms of risk.

So the other options for that are either a medication, and one specific medication called riociguat is approved to relax the lung vessels to try to help that condition, and other medications that we use for other forms of pulmonary hypertension can be useful too. But that medication only takes us so far in somebody who has inoperable or residual thromboembolic disease after prior surgery.

So balloon pulmonary angioplasty is taking techniques as interventional cardiologists we've learned to use in the coronary circulation; putting wires and balloons across areas of narrowing and inflating them to make things less narrow. It has been adapted to lung circulation, where if you can get a wire across one of these narrowing in the lung vessels, then you can put a balloon across it and restore flow to that segment and thereby improve the pulmonary hypertension and the efficiency of the lungs. That technique at an expert center in the last number of years has been shown to have relatively low risk, although not zero, and really good results. So we've been doing quite a lot of that here at the Mayo Clinic in recent years.

I think it's like any newer technique that starts in a small number of centers that get very familiar with it, and then depending on how readily the techniques can be learned and applied, will then spread to other centers. So the nature of this procedure at the present time is that a relatively small number of centers are doing significant numbers of these procedures in the United States, but many centers that have interventional cardiology programs are highly interested in it and are often coming to visit here to try to learn how to do it or visiting a couple of other centers in the country that do a fair amount of this to try to learn the technique.

I would say that it's evolving with it having been in the United States over a decade ago, but maybe not as readily as with current equipment and not maybe quite as successfully. Then, really, the Japanese picked it up and started doing a lot of cases over Japan and demonstrated that it could be done quite effectively. Now the rest of the world is picking it up. Right now in the United States, there's a handful of centers that are doing substantial numbers of procedures, but there's intense interest in it. So I think it will continue to spread across other centers overtime.

We try to emphasize the idea of an expert multidisciplinary approach, where somebody who is really familiar with the options of open surgery, medical therapy and balloon pulmonary angioplasty is involved in each patient's decision making. So the ideal circumstance would be a person who has this diagnosis of chronic thromboembolic pulmonary hypertension is considered at an expert center with regard to whether they're a candidate for open surgery with regard to whether the vessels that are involved are reachable by a surgeon, and [if] that the person is suitable or not.

If they are not found to be suitable for open surgery, then I think they should really be considered for the balloon procedure, because it has, in my sense, a better opportunity of really taking care of the problem with regard to restoring flow to the obstructive segments as supposed to just relaxing the segments that remain open. So we often end up combining the two things of medical therapy to relax the vessels that are still open, plus balloon work. Then seeing whether we can then takeaway the medications if the balloon work is successful enough to improve the flow to a point where they may not be necessary.

On the other hand, in some patients, if the pulmonary hypertension is not too severe when we start with them, then we might do a balloon work alone and never really need the medications, which is nice in a way, because if you're going to use a lifelong medication, then that's going to have potential for side effects and substantial expense, and if you can avoid that, it's a good thing. So I think every patient should be considered for open surgery, balloon work and medication at a center where those decisions can be made properly. If they're not candidates for open surgery, they should be considered for balloon angioplasty.

Honestly, there are relatively very few of the patients with chronic thromboembolic pulmonary hypertension that we've looked at as potential candidates for balloon angioplasty who felt are not candidates for it. Most of the time, there are vessels that could be crossed and the flow can be improved. If the kidneys are really not in good shape, and especially if they're diabetic, then the risk of the contrast we need in order to visualize the vessels well might not be worth it.

So the kind of patient I would not do in would be a diabetic patient with kidney impairment, where you know if you give them quite a lot of contrast, you put them at risk at putting them onto dialysis or something because of renal failure. This is a procedure where you usually need to do multiple sessions. You can't open everything up once. So it might be several sessions of work each one requiring contrast. So if the kidneys are in trouble, you can really hurt them, and maybe those patients are better off just with medical therapy.

I can't emphasize enough how important a V/Q scan is, which is the nuclear scan where you inhale something that shows where the air is going and you have something injected that shows where the blood is going. Those should match like a hand and a glove, and areas that have ventilation but don't have good flow are areas that may be obstructed with clot.

The critical issue is that we're very good at using CT to look for acute clots in somebody who's in the hospital in the emergency room with shortness of breath and have potential for large clots that are easily seen on CT imaging. But the chronic clots that are often pretty far out are easily missed on a CT scan done with thick sections where the number of cross-sections taken isn't close enough together to not miss the webs and bands and maybe reviewed by a radiologist that isn't very familiar with the appearance of chronic old clots that are peripheral.

I've seen multiple patients who were felt to have idiopathic pulmonary arterial hypertension sometimes have been treated for years, and that was on the basis having at a CT that had not shown clots. Then they've come for a second opinion at some point and I’ve said, "Gosh! They never had a ventilation/perfusion lung scan. Let's do that," and find out lo and behold they have chronic thromboembolic pulmonary hypertension, were felt to have idiopathic pulmonary arterial hypertension, because of this issue of the sensitivity of CT scanning being less for findings of chronic clot than a perfusion lung scan.

So if we do a perfusion lung scan that shows evidence of possible clots, then we do an invasive pulmonary angiography, where we put a catheter out in the pulmonary artery and take pictures directly in order to look at the clots in a more distinct way than we can with either CT or with a nuclear perfusion scan. A very important point.

]]>fullEpisode 206 - Robert Frantz, MDRobert Frantz, MD - CTEPHMon, 12 Nov 2018 13:00:00 +0000Robert P. Frantz is the director of the Mayo Pulmonary Hypertension Clinic, which has a robust referral practice of patients with pulmonary arterial hypertension. Dr. Frantz has served as the Mayo site PI on numerous multicenter trials in PH including the majority of trials resulting in FDA approved PH therapies. He also performs complex hemodynamic studies in the catheterization laboratory including right heart catheterizations for patients with pulmonary arterial hypertension and also patients with left heart failure. He also evaluates and manages patients with CTEPH, with particular interest in the role of balloon pulmonary angioplasty.

]]>Robert P. Frantz is the director of the Mayo Pulmonary Hypertension Clinic, which has a robust referral practice of patients with pulmonary arterial hypertension. Dr. Frantz has served as the Mayo site PI on numerous multicenter trials in PH including the majority of trials resulting in FDA approved PH therapies. He also performs complex hemodynamic studies in the catheterization laboratory including right heart catheterizations for patients with pulmonary arterial hypertension and also patients with left heart failure. He also evaluates and manages patients with CTEPH, with particular interest in the role of balloon pulmonary angioplasty.

Canadian caregiver Jennifer Gendron has been working with the pulmonary hypertension community since 2003, when her son Braden was diagnosed at the age of five. She discusses Braden’s PH journey that ultimately led to him having a double-lung transplant at The Hospital for Sick Kids in September 2009.

My name is Jennifer Gendron. I am from Hampton, New Brunswick. I am the mom of a young man [Brayden], who was diagnosed with pulmonary hypertension when he was five, and received a double lung transplant nine years ago. He's now 20, so I guess you would call me a caregiver for life, but I’m. caregiver now of a transplant recipient.

The onset was very subtle. It took a long time for us to get a diagnosis. Looking back now, I guess there were lots of signs, from probably the time he was one or two years old, but very subtle. What really pushed the diagnosis, he was getting short of breath and had a persistent cough. They thought he had asthma. He was diagnosed that same summer with asthma, so he was four-years-old I guess, had just turned five. By fall, things were getting worse. We were finding that he wasn't able to walk very far. He was always complaining he was tired, wanting to be carried around. We had two little boys at the time, twins who were three.

Then in September, things got really worse. What eventually happened, he woke up one morning swollen. We thought he had an allergic reaction. When we took him to the hospital. They did an echocardiogram and discovered that there was fluid around his heart, and he was in congestive heart failure.

Because we live in New Brunswick and there's no Children's Hospital near where we are, they did some initial testing, suspected it was pulmonary hypertension. They were going to send us to Halifax, to the Children's Hospital, to confirm the diagnosis with a heart catheterization, but he was getting progressively worse very quickly, so we ended up being airlifted to Halifax. They were planning on doing a heart catheterization to confirm. He was sick at the time with a virus, so we ended up being there for about a week, before they could actually do the heart catheterization. And when they did, they confirmed the diagnosis.

We were absolutely shocked, because in the matter of a week and a half, we had gone from thinking we had a child with asthma, to being told that he had a terminal illness. Basically we were told at the time, there wasn't much they could do, and that we should take him home, and enjoy the time we had left, was the diagnosis we were given.

At the time, there wasn't very many options for therapy. Flolan (epoprostenol) was the therapy of the time. They were just in the midst of doing trials for sildenafil. We were told by the doctor at this point, who was not a PH Specialist, that we had two options. We could put him on Flolan, and he explained that it was an IV therapy. It was very complicated. He told us we could try this oral therapy, that they were just experimenting with. So that's the route that we went. At the time, I really wasn't ready to just take that at what we were being told. I fought very hard to get to a PH Center. That was in September. It was about three months later before we actually got to Toronto, and were seen in the Pediatric PH Clinic.

By that time, the sildenafil had started. He was improving, so we stayed with that treatment for probably a couple of years after that, but he did end up on IV. In 2005, he was diagnosed, we started on the sildenafil. Couple years later, when things started to deteriorate, by that time Tracleer (bosentan) had come on the scene, so he was started on that. He was on combination with those two oral therapies for another couple of years. But then things continued to progress really quickly. So in 2009, he was started on IV Flolan. He was on, at that point, all three of the drugs.

He's our oldest, so we had two younger boys. They are not quite two years younger than him, who are extremely athletic, and wanted to play every sport that was going. As Brayden got sicker, that was always something that was really a struggle. We were trying to keep a balance in the house, and the let the younger boys do their thing, but it was very hard watching him get sicker and be able to do less and less, while they were running around doing more and more. That was a really tough thing.

In those years, he was the oldest, but the younger two were the ones that looked out for him all the time. They were always running and getting things for him, and helping him. It was almost like the roles were reversed. The little guys were the big brothers, and always taking care of him.

I guess it was probably about a year after he was placed on Flolan, things started to slip again. We had an initial transplant assessment, but then things to stabilize a little bit. We knew transplant was looming, but that we were going to be able to hopefully get a little bit more time. The next year he got sick with pneumonia. After that, he really never seemed to bounce back. We went back to Toronto, and did another assessment. It was time to really start thinking, “The window is closing here, if you're going to pursue transplant. The time is sooner than later.” We went through the whole work up again.

We live in New Brunswick, and being so far from the center added another layer of complication. So in order for him to be listed, we had to move to Toronto. In July of 2009, we went back, did another assessment. He was told that he was a candidate. By that time he was 11, and our younger boys were nine. It was a tough age, because at that stage he's old enough to be a little bit more involved in the whole decision making process. For us, I think that was one of the hardest things. It's this huge decision to make, whether or not you want to go ahead with transplant, and how much of it would be his decision, and how much would be ours.

The day that we made the decision, we were in Toronto waiting to hear back from the hospital, after all of the testing had been done. He had always told us that he hated hockey, and hated sports, and that they were really stupid. I think it was from all the years of being drug around, and not being able to play. So, we're sitting in a restaurant, my cell phone rings, it's the hospital. They said they just wanted to let us know all the tests came back, he was a good candidate, and that he could be listed. I told him, I said, "That was the hospital, and they said that you could go on the transplant list." And he said, "Yes! List me. I can't wait. I'll get some new lungs, and I'll be able to play hockey." At first I lost it, and I said, "You know what? There's our answer right there.” Obviously, there's a lot of things this kid wants to do, that he can't. It's time to make the next move.

We were incredibly lucky, because the whole process went really smooth. That was July. We went back to New Brunswick, packed up our stuff, rented a U-Haul and went back to Toronto. Got to Toronto in August. He was listed. We knew we were there for an indefinite period of time. We figured it would be months. We barely even got settled, a month later he got a call that there were lungs available.

He did incredibly well. A lot of people I know over the years have said to me, "If transplants a cure, basically why doesn't everyone go get a transplant?" It's not the simple, because you're trading one set of issues for another. You never know how it's going to go. You're taking another big risk. He's one of the really lucky ones. He's done incredibly well. He is a very determined, you could say stubborn, young man. They took the ventilator out in the ICU. The very first thing he said to me when he started coming to was, "I told you I could do it." He knew he was going to get through that surgery. Everything was, "I'm going to get out of the hospital. How fast did the last guy get out? Okay, well I'm going to get out sooner. How fast can I do this, and when can I do that?" Every time we would go to an appointment he would ask the doctors, "When can I play hockey?"

He was transplanted in September. His October check-up he asked, and at his November check-up he asked. In January, we went for the follow-up, and he asked the doctor again, "When can I play hockey?" I kept trying to tell him that he wasn't going to play hockey that year, that he had just had major surgery and he would have to wait. The doctor said, "Can you find a league where there's no contact?" And he said, "Yeah, I have." And he said, "Well, then I don't see any reason why you can't play right now." I just about fell off the chair.

Four months after his surgery, he was putting on skates and out on the ice. He played with a bunch of kids that were a lot younger than him. He could hardly stand up. He didn't remember ever even being on skates. He skated when he was little, but he didn't remember. So at 11-years-old, he started playing hockey for the first time ever. Then it was just one thing after another. It was, “what can I try next?” Just last weekend, he celebrated - we call it his “re-birthday.” So nine years.

Physically he's done phenomenally well. Emotionally it's been a struggle a lot of the time. I think a lot of it was the age, being a pre-teen, being in middle school, and trying to adjust after being a kid who was sick his whole life, and what he remembered, and never having been able to do the things that normal kids his age could do. He really never had a time that he was super stable. A lot of the patients that I meet, do really, really well. Brayden never really got that well, that he was able to do a lot of things.

I think never having been able to be a normal kid, I say that loosely, but he just struggled with how to fit in. Even the dynamic in our house. We went from, being the guy who was always looked after by his brothers, and by his dad, and by [me] to being a kid who, "Well, you don't really need us to run and get that for you, because you're able to do that now." It was really hard for him to adjust to just being an average guy. The last few years, that's been the struggle. Not so much the physical side of things, it's been more of the emotional/social side of things that have been difficult. It's was super tough. Never really having to let him go do things, because he wasn't really able. So having to try and let go, and let him take the normal steps. It was tough. It was a big adjustment.

I guess for me, a lot of it was maybe not wanting him to do things. The hockey for example, all these things thinking, "Oh my gosh. Is he going to get hurt? Is he going to be okay?" I remember him saying to me one time that I was worrying over something he wanted to do, "Mom, I didn't go through all of this surgery and all of this stuff so that I could just sit back and watch other people. I want to live my life."

So, when you hear something like that, it's like, "All right. You know what? It's true. He didn't go through all that, to be held back from things he wanted to do." So it's just like, "All right, you got to just let go." It's still hard. I mean, today it's hard. He's 20, so it's getting even harder, because now he's an adult, and he's out there wanting to do things. We have let go a little bit more. He doesn't want to hear his mom say, "Did you take your pills today? Have you done your pulmonary function test?" He doesn't want to listen to that right now, but it's hard to not still want to look after him.

Canadian caregiver Jennifer Gendron has been working with the pulmonary hypertension community since 2003, when her son Braden was diagnosed at the age of five. She discusses Braden’s PH journey that ultimately led to him having a double-lung transplant at The Hospital for Sick Kids in September 2009.

My name is Jennifer Gendron. I am from Hampton, New Brunswick. I am the mom of a young man [Brayden], who was diagnosed with pulmonary hypertension when he was five, and received a double lung transplant nine years ago. He's now 20, so I guess you would call me a caregiver for life, but I’m. caregiver now of a transplant recipient.

The onset was very subtle. It took a long time for us to get a diagnosis. Looking back now, I guess there were lots of signs, from probably the time he was one or two years old, but very subtle. What really pushed the diagnosis, he was getting short of breath and had a persistent cough. They thought he had asthma. He was diagnosed that same summer with asthma, so he was four-years-old I guess, had just turned five. By fall, things were getting worse. We were finding that he wasn't able to walk very far. He was always complaining he was tired, wanting to be carried around. We had two little boys at the time, twins who were three.

Then in September, things got really worse. What eventually happened, he woke up one morning swollen. We thought he had an allergic reaction. When we took him to the hospital. They did an echocardiogram and discovered that there was fluid around his heart, and he was in congestive heart failure.

Because we live in New Brunswick and there's no Children's Hospital near where we are, they did some initial testing, suspected it was pulmonary hypertension. They were going to send us to Halifax, to the Children's Hospital, to confirm the diagnosis with a heart catheterization, but he was getting progressively worse very quickly, so we ended up being airlifted to Halifax. They were planning on doing a heart catheterization to confirm. He was sick at the time with a virus, so we ended up being there for about a week, before they could actually do the heart catheterization. And when they did, they confirmed the diagnosis.

We were absolutely shocked, because in the matter of a week and a half, we had gone from thinking we had a child with asthma, to being told that he had a terminal illness. Basically we were told at the time, there wasn't much they could do, and that we should take him home, and enjoy the time we had left, was the diagnosis we were given.

At the time, there wasn't very many options for therapy. Flolan (epoprostenol) was the therapy of the time. They were just in the midst of doing trials for sildenafil. We were told by the doctor at this point, who was not a PH Specialist, that we had two options. We could put him on Flolan, and he explained that it was an IV therapy. It was very complicated. He told us we could try this oral therapy, that they were just experimenting with. So that's the route that we went. At the time, I really wasn't ready to just take that at what we were being told. I fought very hard to get to a PH Center. That was in September. It was about three months later before we actually got to Toronto, and were seen in the Pediatric PH Clinic.

By that time, the sildenafil had started. He was improving, so we stayed with that treatment for probably a couple of years after that, but he did end up on IV. In 2005, he was diagnosed, we started on the sildenafil. Couple years later, when things started to deteriorate, by that time Tracleer (bosentan) had come on the scene, so he was started on that. He was on combination with those two oral therapies for another couple of years. But then things continued to progress really quickly. So in 2009, he was started on IV Flolan. He was on, at that point, all three of the drugs. He's our oldest, so we had two younger boys. They are not quite two years younger than him, who are extremely athletic, and wanted to play every sport that was going. As Brayden got sicker, that was always something that was really a struggle. We were trying to keep a balance in the house, and the let the younger boys do their thing, but it was very hard watching him get sicker and be able to do less and less, while they were running around doing more and more. That was a really tough thing.

In those years, he was the oldest, but the younger two were the ones that looked out for him all the time. They were always running and getting things for him, and helping him. It was almost like the roles were reversed. The little guys were the big brothers, and always taking care of him.

I guess it was probably about a year after he was placed on Flolan, things started to slip again. We had an initial transplant assessment, but then things to stabilize a little bit. We knew transplant was looming, but that we were going to be able to hopefully get a little bit more time. The next year he got sick with pneumonia. After that, he really never seemed to bounce back. We went back to Toronto, and did another assessment. It was time to really start thinking, “The window is closing here, if you're going to pursue transplant. The time is sooner than later.” We went through the whole work up again.

We live in New Brunswick, and being so far from the center added another layer of complication. So in order for him to be listed, we had to move to Toronto. In July of 2009, we went back, did another assessment. He was told that he was a candidate. By that time he was 11, and our younger boys were nine. It was a tough age, because at that stage he's old enough to be a little bit more involved in the whole decision making process. For us, I think that was one of the hardest things. It's this huge decision to make, whether or not you want to go ahead with transplant, and how much of it would be his decision, and how much would be ours.

The day that we made the decision, we were in Toronto waiting to hear back from the hospital, after all of the testing had been done. He had always told us that he hated hockey, and hated sports, and that they were really stupid. I think it was from all the years of being drug around, and not being able to play. So, we're sitting in a restaurant, my cell phone rings, it's the hospital. They said they just wanted to let us know all the tests came back, he was a good candidate, and that he could be listed. I told him, I said, "That was the hospital, and they said that you could go on the transplant list." And he said, "Yes! List me. I can't wait. I'll get some new lungs, and I'll be able to play hockey." At first I lost it, and I said, "You know what? There's our answer right there.” Obviously, there's a lot of things this kid wants to do, that he can't. It's time to make the next move.

We were incredibly lucky, because the whole process went really smooth. That was July. We went back to New Brunswick, packed up our stuff, rented a U-Haul and went back to Toronto. Got to Toronto in August. He was listed. We knew we were there for an indefinite period of time. We figured it would be months. We barely even got settled, a month later he got a call that there were lungs available.

He did incredibly well. A lot of people I know over the years have said to me, "If transplants a cure, basically why doesn't everyone go get a transplant?" It's not the simple, because you're trading one set of issues for another. You never know how it's going to go. You're taking another big risk. He's one of the really lucky ones. He's done incredibly well. He is a very determined, you could say stubborn, young man. They took the ventilator out in the ICU. The very first thing he said to me when he started coming to was, "I told you I could do it." He knew he was going to get through that surgery. Everything was, "I'm going to get out of the hospital. How fast did the last guy get out? Okay, well I'm going to get out sooner. How fast can I do this, and when can I do that?" Every time we would go to an appointment he would ask the doctors, "When can I play hockey?"

He was transplanted in September. His October check-up he asked, and at his November check-up he asked. In January, we went for the follow-up, and he asked the doctor again, "When can I play hockey?" I kept trying to tell him that he wasn't going to play hockey that year, that he had just had major surgery and he would have to wait. The doctor said, "Can you find a league where there's no contact?" And he said, "Yeah, I have." And he said, "Well, then I don't see any reason why you can't play right now." I just about fell off the chair.

Four months after his surgery, he was putting on skates and out on the ice. He played with a bunch of kids that were a lot younger than him. He could hardly stand up. He didn't remember ever even being on skates. He skated when he was little, but he didn't remember. So at 11-years-old, he started playing hockey for the first time ever. Then it was just one thing after another. It was, “what can I try next?” Just last weekend, he celebrated - we call it his “re-birthday.” So nine years.

Physically he's done phenomenally well. Emotionally it's been a struggle a lot of the time. I think a lot of it was the age, being a pre-teen, being in middle school, and trying to adjust after being a kid who was sick his whole life, and what he remembered, and never having been able to do the things that normal kids his age could do. He really never had a time that he was super stable. A lot of the patients that I meet, do really, really well. Brayden never really got that well, that he was able to do a lot of things.

I think never having been able to be a normal kid, I say that loosely, but he just struggled with how to fit in. Even the dynamic in our house. We went from, being the guy who was always looked after by his brothers, and by his dad, and by [me] to being a kid who, "Well, you don't really need us to run and get that for you, because you're able to do that now." It was really hard for him to adjust to just being an average guy. The last few years, that's been the struggle. Not so much the physical side of things, it's been more of the emotional/social side of things that have been difficult. It's was super tough. Never really having to let him go do things, because he wasn't really able. So having to try and let go, and let him take the normal steps. It was tough. It was a big adjustment.

I guess for me, a lot of it was maybe not wanting him to do things. The hockey for example, all these things thinking, "Oh my gosh. Is he going to get hurt? Is he going to be okay?" I remember him saying to me one time that I was worrying over something he wanted to do, "Mom, I didn't go through all of this surgery and all of this stuff so that I could just sit back and watch other people. I want to live my life."

So, when you hear something like that, it's like, "All right. You know what? It's true. He didn't go through all that, to be held back from things he wanted to do." So it's just like, "All right, you got to just let go." It's still hard. I mean, today it's hard. He's 20, so it's getting even harder, because now he's an adult, and he's out there wanting to do things. We have let go a little bit more. He doesn't want to hear his mom say, "Did you take your pills today? Have you done your pulmonary function test?" He doesn't want to listen to that right now, but it's hard to not still want to look after him.

]]>fullEpisode 205 - Jennifer GendronJennifer Gendron - Pulmonary HypertensionThu, 08 Nov 2018 13:00:00 +0000Canadian caregiver Jennifer Gendron has been working with the pulmonary hypertension community since 2003, when her son Braden was diagnosed at the age of five. She discusses Braden’s PH journey that ultimately led to him having a double-lung transplant at The Hospital for Sick Kids in September 2009.

]]>Canadian caregiver Jennifer Gendron has been working with the pulmonary hypertension community since 2003, when her son Braden was diagnosed at the age of five. She discusses Braden’s PH journey that ultimately led to him having a double-lung transplant at The Hospital for Sick Kids in September 2009.

Janette Reyes, NP has provided comprehensive care for children with Pulmonary Hypertension at the Hospital for Sick Children since the development of the Pulmonary Hypertension Service in the year 2000. She is involved in research and strives to improve pediatric patients’ quality of life. Janette discusses Child to Adult Transition Guidelines specific to the pulmonary hypertension patient population.

I'm Janette Reyes. I'm a nurse practitioner, and I work at the Hospital for Sick Children in the cardiac program, and I care for patients with pulmonary hypertension from age zero to just right before they turn 18 years of age when they're admitted as an inpatient and mostly when they visit the clinics.

Back in 2000, I did see the need for a transition program for our pulmonary hypertension patients, specifically from one pulmonary hypertension center to an adult pulmonary hypertension center. In 2005, along with a transition program at the Hospital for Sick Children, I developed a workshop where the patients who are 17 years of age, who are ready to transition into the adult world, I would hold a half day workshop helping parents and those patients learn about what's on the other side. Then I would have the doctor in the adult center come over, introduce himself, put a face to the doctor that they'll be seeing. Parents and patients would ask some questions and then we would go over and I would give them a tour of the center.

The patients really found that valuable, so we've been doing that since, as well those patients who can't attend the workshop I do a one-on-one individual session. Parents and the children completed evaluations after each session and I put together a little survey. Prior to the transition, the parents were actually more anxious and nervous about their child going to the adult world. But then after the transition, the parents were comfortable. It was more the children that kind of [experienced] their anxiety rise a little bit. I guess knowing now seeing what's on the other side. However, when I do speak to some of the patients and families, they did say the transition workshop really helped them and as well in terms of preparing and just knowing what to expect really was helpful to them.

At age 14 is when we start preparing. We start the preparation talking about their disease more, making sure they understand why they're in the clinic. As they get older, age 16 and up, they start to become more responsible with their medication. We encourage them to perhaps renew their medication, their prescription and as well we start talking about college or university, what to do, where to go, and we help them create a passport which includes their diagnosis, medications, doctors, our contact information, the new doctor's information, the hospital, just so that if ever they go to [the E.R.], the doctor would have that information. They found that helpful as well. I think that helps.

Children are different, right? Each child is at a different cognitive stage level, so some are more mature than others and we take particular note of that in regards to the speed of the transition. I think every family's different. You will have children that are more dependent on the parent because of the helicopter-type of behavior from the parents. But because we do prepare the patients and expect that in families, particularly with me, when I see the families in clinic I do at age 14 start to give them an idea (the parents particularly) “Start moving away from the care and allow your child to be more independent and let them know that at the end it's really for their good and for their benefit.” Then that sort of helps them that they don't feel that we're trying to separate them. We're just trying to make them independent because for whatever reason if the parent isn't there, then at least the child is going to have some control and independence in their care. That process is getting better, but we do have the challenges in that situation and I think with the awareness of the adult centers having these type of families going into their centers, they're more prepared.

Now it's more of a transition, so the adult world aren't just going to start their treatment as an adult for the patient, it's more of a transition. In the beginning, they will include the parent and then slowly start to speak to the patient on their own. Teenagers, young teenagers they're so laid back. They actually don't think too far off into the future, so whenever I would ask them, "So, are you nervous going to the adult world?" It's always like, "No, it's going to be fine." Just like the survey, or the evaluation that I found, they weren't as anxious as the parents. If anything, I do encourage them to think about issues that they may have, questions that potentially maybe they're not thinking at the time, but it's going to be issues as they get older. In particular, sexuality, protection, issues with going out into the workforce. Again, like as I mentioned earlier, university. Those things, even though if they don't tell me, I know that's going to be issues that could certainly contribute to their anxiety.

We kind of make sure that they can talk to me about that and I help them with the transition by giving them the information and resources that are available out there. The reason why this transition is happening is because certainly there are more and more targeted vasodilators that are being approved by the FDA and the Ministry of Health in Canada, so these children are living longer and it's great to see. Right now where we're at, even though this transition program was started early in the 2000s, I find that actually now it's still even a stronger issue that needs to be addressed, because there are more children that are transitioning into the adult centers. We need to get, as well now, the adult centers involved in this transition and to understand these young adults. They're not completely adults yet, they're in transition.

Janette Reyes, NP has provided comprehensive care for children with Pulmonary Hypertension at the Hospital for Sick Children since the development of the Pulmonary Hypertension Service in the year 2000. She is involved in research and strives to improve pediatric patients’ quality of life. Janette discusses Child to Adult Transition Guidelines specific to the pulmonary hypertension patient population.

I'm Janette Reyes. I'm a nurse practitioner, and I work at the Hospital for Sick Children in the cardiac program, and I care for patients with pulmonary hypertension from age zero to just right before they turn 18 years of age when they're admitted as an inpatient and mostly when they visit the clinics.

Back in 2000, I did see the need for a transition program for our pulmonary hypertension patients, specifically from one pulmonary hypertension center to an adult pulmonary hypertension center. In 2005, along with a transition program at the Hospital for Sick Children, I developed a workshop where the patients who are 17 years of age, who are ready to transition into the adult world, I would hold a half day workshop helping parents and those patients learn about what's on the other side. Then I would have the doctor in the adult center come over, introduce himself, put a face to the doctor that they'll be seeing. Parents and patients would ask some questions and then we would go over and I would give them a tour of the center.

The patients really found that valuable, so we've been doing that since, as well those patients who can't attend the workshop I do a one-on-one individual session. Parents and the children completed evaluations after each session and I put together a little survey. Prior to the transition, the parents were actually more anxious and nervous about their child going to the adult world. But then after the transition, the parents were comfortable. It was more the children that kind of [experienced] their anxiety rise a little bit. I guess knowing now seeing what's on the other side. However, when I do speak to some of the patients and families, they did say the transition workshop really helped them and as well in terms of preparing and just knowing what to expect really was helpful to them.

At age 14 is when we start preparing. We start the preparation talking about their disease more, making sure they understand why they're in the clinic. As they get older, age 16 and up, they start to become more responsible with their medication. We encourage them to perhaps renew their medication, their prescription and as well we start talking about college or university, what to do, where to go, and we help them create a passport which includes their diagnosis, medications, doctors, our contact information, the new doctor's information, the hospital, just so that if ever they go to [the E.R.], the doctor would have that information. They found that helpful as well. I think that helps.

Children are different, right? Each child is at a different cognitive stage level, so some are more mature than others and we take particular note of that in regards to the speed of the transition. I think every family's different. You will have children that are more dependent on the parent because of the helicopter-type of behavior from the parents. But because we do prepare the patients and expect that in families, particularly with me, when I see the families in clinic I do at age 14 start to give them an idea (the parents particularly) “Start moving away from the care and allow your child to be more independent and let them know that at the end it's really for their good and for their benefit.” Then that sort of helps them that they don't feel that we're trying to separate them. We're just trying to make them independent because for whatever reason if the parent isn't there, then at least the child is going to have some control and independence in their care. That process is getting better, but we do have the challenges in that situation and I think with the awareness of the adult centers having these type of families going into their centers, they're more prepared.

Now it's more of a transition, so the adult world aren't just going to start their treatment as an adult for the patient, it's more of a transition. In the beginning, they will include the parent and then slowly start to speak to the patient on their own. Teenagers, young teenagers they're so laid back. They actually don't think too far off into the future, so whenever I would ask them, "So, are you nervous going to the adult world?" It's always like, "No, it's going to be fine." Just like the survey, or the evaluation that I found, they weren't as anxious as the parents. If anything, I do encourage them to think about issues that they may have, questions that potentially maybe they're not thinking at the time, but it's going to be issues as they get older. In particular, sexuality, protection, issues with going out into the workforce. Again, like as I mentioned earlier, university. Those things, even though if they don't tell me, I know that's going to be issues that could certainly contribute to their anxiety.

We kind of make sure that they can talk to me about that and I help them with the transition by giving them the information and resources that are available out there. The reason why this transition is happening is because certainly there are more and more targeted vasodilators that are being approved by the FDA and the Ministry of Health in Canada, so these children are living longer and it's great to see. Right now where we're at, even though this transition program was started early in the 2000s, I find that actually now it's still even a stronger issue that needs to be addressed, because there are more children that are transitioning into the adult centers. We need to get, as well now, the adult centers involved in this transition and to understand these young adults. They're not completely adults yet, they're in transition.

]]>fullEpisode 204 - Janette Reyes, NPJanette Reyes - Pulmonary HypertensionMon, 05 Nov 2018 13:00:00 +0000Janette Reyes, NP has provided comprehensive care for children with Pulmonary Hypertension at the Hospital for Sick Children since the development of the Pulmonary Hypertension Service in the year 2000. She is involved in research and strives to improve pediatric patients’ quality of life. Janette discusses Child to Adult Transition Guidelines specific to the pulmonary hypertension patient population.

]]>Janette Reyes, NP has provided comprehensive care for children with Pulmonary Hypertension at the Hospital for Sick Children since the development of the Pulmonary Hypertension Service in the year 2000. She is involved in research and strives to improve pediatric patients’ quality of life. Janette discusses Child to Adult Transition Guidelines specific to the pulmonary hypertension patient population.

]]>06:34cleanblood,research,heart,pediatric,awareness,therapies,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdiseaseJanette Reyes, NP has provided comprehensive care for children with Pulmonary Hypertension at the Hospital for Sick Children since the development of the Pulmonary Hypertension Service in the year 2000. Janette discusses Child to Adult Transition Guidelines specific to the pulmonary hypertension patient population.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us @phaware. @antidote_me @phacanada #phaware #phawareMD @SickKidsNews35fullphaware global associationSam Bowker and Ken Porter - phaware® interview 203Fri, 02 Nov 2018 12:00:00 +0000

Sam Bowker is a pulmonary hypertension patient on triple therapy from Canada. Ken Porter is her partner and caregiver. They discuss Sam’s diagnosis and creative ways they work together to manage her PH treatment regimen.

Sam: My name is Sam Bowker. I'm a patient at the Vancouver PH Clinic. But I live in Victoria. I was diagnosed with pulmonary hypertension almost three years ago. So my diagnosis date is November.

Ken: My name is Ken Porter. I'm Sam's partner. We've been together for eight, nine years now. I’m supporting her through this, and yeah, it's been a ride.

Sam: I was running a race series, and was in super-good health. I'm a teacher, so I took a group of students to France. And while I was there, I started to notice that I was having this weird chest pain. Which was strange for me because I was super active. I wasn't really expecting that. And then I came home and it just got progressively worse. And worse. So I went to the doctor and he told me I had anxiety. I was like, "Well, I don't really feel anxious, and I'm getting chest pain when I walk upstairs. So it's not like that's causing a lot of anxiety." So, after visiting him quite a few times and kind of harassing him, he sent me to a cardiologist, and the cardiologist did a bunch of tests. He said I was in really good health. I was insistent that something was very wrong. And so he sent me to do an stress echo, so I had to ride a bike while they did the echocardiogram. As soon as the tech touched the wand to my chest, she said it's pulmonary hypertension. Then she left the room. I was like, "OK..?." And then when she came back, I said, "What's pulmonary hypertension?" And she said, "I'd rather you talk to your cardiologist about it." And then she said, "Don't Google it." So of course, I did Google it. And we were pretty freaked out.

Ken: I remember that day. Sam sent me a text, said pulmonary hypertension, and of course I immediately Googled it. And it was a shock. I was at work; I couldn't work for the rest of the day. That evening, we were both in shambles, trying to figure out what this meant for our lives, and where we were going to go from there. And we hadn't even talked to a specialist yet. That was just the opinion of a tech talking to Sam, and then us trying to figure through it. It was an adjustment, just trying to figure out what this was going to mean for us, and we got over the initial shock after a few days. Once we were able to talk to a specialist, we realized, "Hey, this is an evolving area. This is an evolving science. There's a lot of really good things happening." All the stuff on the internet that we could find was likely already dated, which was good, because it was really scary. Who knows what the prognosis was going to be, because of the evolution of medicine and technology.

Sam: I finally got to the clinic in Vancouver, and they did a right heart catheterization and right away, I wasn't really given another option. I was told right away I was going to go on IV meds. Because I was really sick. But then, of course, they started me on oral meds as well, because they're trying to get all of the different pathways. We tried those for a few months, and it wasn't really having as positive an effect on my pressures as they liked. And so I started on a third therapy. So I'm on the tri-therapy.

Ken: Initially, we had a series of sessions, when they put Sam's line in, the rules state you can't leave the hospital until you can show that you can mix the medicine. As we were going through the various workshops and understanding how to do it, we once again, massively overwhelmed. "How are we going to live life, how're we going to mix meds and be able to do anything else?" We recognized very quickly, that it's going to be a team effort. It's going to be an adjustment. We were able to show, I think in a matter of days, that we could effectively do this. We were competent. So they said, "You could leave the hospital." We left as fast as we could, because we wanted to go home. I think we were in the hospital for a total of a week. When we got home, and ever since, over the course of the last three years, we've divvied up mixing the medicine. And we've gotten really good at it. You think most people spend some time on the couch watching a movie or TV or sports. So now, instead of just lying on the couch while watching TV, movie, or sports, we'll mix medicine while we're doing that. So we've managed to make it a part of our lives. And yes, it's something additional we have to do, but we've managed to make it work. And we've even noticed that sometimes we won't have the TV on. So, it provides an opportunity for us to bond even further. We can chat while we're making medicine.

Sam: It's like a bizarre date.

Ken: Yeah, it's a bizarre date. Traditionally, we wouldn't have maybe sat down and chatted while we were doing something like this. So, yeah, there's positives that come out of it. Yeah, it's something additional we have to do in life. But we make it work.

Sam: I think that there's a trust that has to happen there, because you really are, when you're putting the cassette on you. It's your life source, right? So you have to trust your partner and know that what they're doing is what you would do. I'm really lucky and that Ken's really supportive, and I have that trust with him. It's really helpful. It's made our lives so that I can do things sometimes that I wouldn't normally ever be able to do. For example, I did my yoga teacher training in, so Ken had to mix my meds every weekend because I wasn't there to do it. It's super helpful to have someone else who can mix meds for me.

Ken: The last two summers, Sam and I have been fortunate enough to travel to Europe. So we figured out how to make this work when we're traveling, too. We will get AirB&Bs that have fridges. We'll set up a little thing on the kitchen table. We'll make the medicine, and if we both do it together, we do it fast. It gives us more time to go out and enjoy the sights. We've gotten really good at it.

Sam: I was leave for three months while I got adjusted to doing all of the medication business, and then I went back to work. Originally, I went back to work full time. I was a schoolteacher, So, full time working with the kids, I found that I was just coming home and I was totally exhausted. My team is incredibly supportive. They really focus on our quality of life. Anything that I'm like, "I want to do this," they're like, "Awesome. Do it." They want to see us doing what we love. And I worked really hard to become a teacher. It's something I really wanted to do, I was really passionate about it. So I didn't really want to give it up. So we've managed to make it work.

Ken: I'm an athlete, and so I play a tremendous amount of ice hockey. I play ice hockey with a close-knit network of friends that I've had since elementary school, which is insane. It gives me an avenue to go out and exert energy, and just come back recharged. I'll play ice hockey two, three times a week. And if I'm feeling down, maybe I also go to the gym. I just remain really active. Sam and I as a collective also, we've engaged in meditation, which we found has just been a fantastic tool to use to center ourselves and stay calm. I wouldn't say nightly, but we're getting pretty good at doing it probably once every two days or so. Engaging in some form of a meditation practice. And one other thing we've done that has been really a lot more beneficial than I could ever have anticipated. We went out and bought two kittens. And just having two cats around the house that are really cute and want to cuddle. We could feel it change the energy in the house, the demeanor. It was a really valuable tool. We had heard from some people that pets are a really good thing to have with the condition. It's exceeded our expectations what the two kittens did for us.

Sam: I would also say, do whatever you can to stay positive. I think that that really improves your quality of life. It's hard sometimes. You get dragged down by all the things that happen in our community, and all the people that you get close to. And then there's a lot of loss.

Ken: I would also add two things. A, recognize that it's an emotional roller coaster. You're going to have perhaps days where there's bad news, or you're going to be overwhelmed by some of the approaches you may have to take. But honestly, it gets better. You learn to make it a new normal, and to make the best of life. And I think it's important just to recognize that. When there can be times when you're really way down, you will get back up, and just once, like what Sam says, stay positive. We're living in such an exciting world right now, where the evolution of medicine and technology is mind boggling. Back in the '80s, when someone got sick, they may be faced with the stark reality that there's not going to be something new that's going to come in the near future. And they [just had] to deal with that. I am just so excited that we live in a world right now where a new medicine and new approach and new technology could come out in the next three, four, six months. Who knows? That could resolve all kinds of issues. So, when it comes to pulmonary hypertension, I am very optimistic. I know there's lots of money, lots of resources going into trying to look for a cure, and ways to better manage this condition. A year from now, who knows? You have to have that positive outlook, and recognize that there could be a solution around the corner. Hold out hope for that, for sure.

Sam Bowker is a pulmonary hypertension patient on triple therapy from Canada. Ken Porter is her partner and caregiver. They discuss Sam’s diagnosis and creative ways they work together to manage her PH treatment regimen.

Sam: My name is Sam Bowker. I'm a patient at the Vancouver PH Clinic. But I live in Victoria. I was diagnosed with pulmonary hypertension almost three years ago. So my diagnosis date is November.

Ken: My name is Ken Porter. I'm Sam's partner. We've been together for eight, nine years now. I’m supporting her through this, and yeah, it's been a ride.

Sam: I was running a race series, and was in super-good health. I'm a teacher, so I took a group of students to France. And while I was there, I started to notice that I was having this weird chest pain. Which was strange for me because I was super active. I wasn't really expecting that. And then I came home and it just got progressively worse. And worse. So I went to the doctor and he told me I had anxiety. I was like, "Well, I don't really feel anxious, and I'm getting chest pain when I walk upstairs. So it's not like that's causing a lot of anxiety." So, after visiting him quite a few times and kind of harassing him, he sent me to a cardiologist, and the cardiologist did a bunch of tests. He said I was in really good health. I was insistent that something was very wrong. And so he sent me to do an stress echo, so I had to ride a bike while they did the echocardiogram. As soon as the tech touched the wand to my chest, she said it's pulmonary hypertension. Then she left the room. I was like, "OK..?." And then when she came back, I said, "What's pulmonary hypertension?" And she said, "I'd rather you talk to your cardiologist about it." And then she said, "Don't Google it." So of course, I did Google it. And we were pretty freaked out.

Ken: I remember that day. Sam sent me a text, said pulmonary hypertension, and of course I immediately Googled it. And it was a shock. I was at work; I couldn't work for the rest of the day. That evening, we were both in shambles, trying to figure out what this meant for our lives, and where we were going to go from there. And we hadn't even talked to a specialist yet. That was just the opinion of a tech talking to Sam, and then us trying to figure through it. It was an adjustment, just trying to figure out what this was going to mean for us, and we got over the initial shock after a few days. Once we were able to talk to a specialist, we realized, "Hey, this is an evolving area. This is an evolving science. There's a lot of really good things happening." All the stuff on the internet that we could find was likely already dated, which was good, because it was really scary. Who knows what the prognosis was going to be, because of the evolution of medicine and technology.

Sam: I finally got to the clinic in Vancouver, and they did a right heart catheterization and right away, I wasn't really given another option. I was told right away I was going to go on IV meds. Because I was really sick. But then, of course, they started me on oral meds as well, because they're trying to get all of the different pathways. We tried those for a few months, and it wasn't really having as positive an effect on my pressures as they liked. And so I started on a third therapy. So I'm on the tri-therapy.

Ken: Initially, we had a series of sessions, when they put Sam's line in, the rules state you can't leave the hospital until you can show that you can mix the medicine. As we were going through the various workshops and understanding how to do it, we once again, massively overwhelmed. "How are we going to live life, how're we going to mix meds and be able to do anything else?" We recognized very quickly, that it's going to be a team effort. It's going to be an adjustment. We were able to show, I think in a matter of days, that we could effectively do this. We were competent. So they said, "You could leave the hospital." We left as fast as we could, because we wanted to go home. I think we were in the hospital for a total of a week. When we got home, and ever since, over the course of the last three years, we've divvied up mixing the medicine. And we've gotten really good at it. You think most people spend some time on the couch watching a movie or TV or sports. So now, instead of just lying on the couch while watching TV, movie, or sports, we'll mix medicine while we're doing that. So we've managed to make it a part of our lives. And yes, it's something additional we have to do, but we've managed to make it work. And we've even noticed that sometimes we won't have the TV on. So, it provides an opportunity for us to bond even further. We can chat while we're making medicine.

Sam: It's like a bizarre date.

Ken: Yeah, it's a bizarre date. Traditionally, we wouldn't have maybe sat down and chatted while we were doing something like this. So, yeah, there's positives that come out of it. Yeah, it's something additional we have to do in life. But we make it work.

Sam: I think that there's a trust that has to happen there, because you really are, when you're putting the cassette on you. It's your life source, right? So you have to trust your partner and know that what they're doing is what you would do. I'm really lucky and that Ken's really supportive, and I have that trust with him. It's really helpful. It's made our lives so that I can do things sometimes that I wouldn't normally ever be able to do. For example, I did my yoga teacher training in, so Ken had to mix my meds every weekend because I wasn't there to do it. It's super helpful to have someone else who can mix meds for me.

Ken: The last two summers, Sam and I have been fortunate enough to travel to Europe. So we figured out how to make this work when we're traveling, too. We will get AirB&Bs that have fridges. We'll set up a little thing on the kitchen table. We'll make the medicine, and if we both do it together, we do it fast. It gives us more time to go out and enjoy the sights. We've gotten really good at it.

Sam: I was leave for three months while I got adjusted to doing all of the medication business, and then I went back to work. Originally, I went back to work full time. I was a schoolteacher, So, full time working with the kids, I found that I was just coming home and I was totally exhausted. My team is incredibly supportive. They really focus on our quality of life. Anything that I'm like, "I want to do this," they're like, "Awesome. Do it." They want to see us doing what we love. And I worked really hard to become a teacher. It's something I really wanted to do, I was really passionate about it. So I didn't really want to give it up. So we've managed to make it work.

Ken: I'm an athlete, and so I play a tremendous amount of ice hockey. I play ice hockey with a close-knit network of friends that I've had since elementary school, which is insane. It gives me an avenue to go out and exert energy, and just come back recharged. I'll play ice hockey two, three times a week. And if I'm feeling down, maybe I also go to the gym. I just remain really active. Sam and I as a collective also, we've engaged in meditation, which we found has just been a fantastic tool to use to center ourselves and stay calm. I wouldn't say nightly, but we're getting pretty good at doing it probably once every two days or so. Engaging in some form of a meditation practice. And one other thing we've done that has been really a lot more beneficial than I could ever have anticipated. We went out and bought two kittens. And just having two cats around the house that are really cute and want to cuddle. We could feel it change the energy in the house, the demeanor. It was a really valuable tool. We had heard from some people that pets are a really good thing to have with the condition. It's exceeded our expectations what the two kittens did for us.

Sam: I would also say, do whatever you can to stay positive. I think that that really improves your quality of life. It's hard sometimes. You get dragged down by all the things that happen in our community, and all the people that you get close to. And then there's a lot of loss.

Ken: I would also add two things. A, recognize that it's an emotional roller coaster. You're going to have perhaps days where there's bad news, or you're going to be overwhelmed by some of the approaches you may have to take. But honestly, it gets better. You learn to make it a new normal, and to make the best of life. And I think it's important just to recognize that. When there can be times when you're really way down, you will get back up, and just once, like what Sam says, stay positive. We're living in such an exciting world right now, where the evolution of medicine and technology is mind boggling. Back in the '80s, when someone got sick, they may be faced with the stark reality that there's not going to be something new that's going to come in the near future. And they [just had] to deal with that. I am just so excited that we live in a world right now where a new medicine and new approach and new technology could come out in the next three, four, six months. Who knows? That could resolve all kinds of issues. So, when it comes to pulmonary hypertension, I am very optimistic. I know there's lots of money, lots of resources going into trying to look for a cure, and ways to better manage this condition. A year from now, who knows? You have to have that positive outlook, and recognize that there could be a solution around the corner. Hold out hope for that, for sure.

]]>Sam Bowker is a pulmonary hypertension patient on triple therapy from Canada. Ken Porter is her partner and caregiver. They discuss Sam’s diagnosis and creative ways they work together to manage her PH treatment regimen.

]]>08:42cleanblood,canada,research,heart,awareness,asthma,iv,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdisease,subqSam Bowker is a pulmonary hypertension patient on triple therapy from Canada. Ken Porter is her partner and caregiver. They discuss Sam’s diagnosis and creative ways they work together to manage her PH treatment regiment.
Learn more about pulmonary hypertension trials a www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware. Engage for a cure: www.phaware.global/donate #phaware #YoungwithPAH #LifeInPurple #PHUnited @PHACanada @antidote_me 34fullphaware global associationNatalie Roy - phaware® interview 202Tue, 30 Oct 2018 12:00:00 +0000

Canadian Natalie Roy is a former pulmonary hypertension patient who received a double-lung transplant in September 2017. She discusses having an identity crisis post PH.

My name is Natalie Roy. I live in Vancouver, British Columbia, Canada. I was diagnosed with, what was called at the time, primary pulmonary hypertension in 2005 when I was 20. Currently, I am one year past new lungs. I just celebrated my one-year lungiversary.

I just started fainting. I started noticing that I was having a hard time walking up hills. At the time, I just wanted to avoid it. I think I was scared, but I was like, "Oh, I'm just dehydrated, or I need a nap, or maybe I haven't eaten enough today." I just was in denial and altered my life patterns to better fit not walking up hills or walking upstairs or whatever.

I fainted walking up a set of stairs. And somebody had called an ambulance. I guess my lips had gone blue, and they couldn't wake me up for over 10 minutes. So I got diagnosed in the emergency room with six months to live, at the time without treatment.

I got on some oral treatment, and made a life change, and moved to Alberta and went to school for chef's school. I did really well for the first five years on just straight oral treatment. And then I went to Australia for a year. And when I came back, I started fainting again. So they put me on a secondary oral. That lasted about two years, and then I started having symptoms and went on IV medication for five years. And got listed for a double-lung transplant November 1st, 2015. And received my lungs September 7th, 2017.

I had always told myself when my team tells me it's time, it doesn't matter if I feel ready or not, then I'll just do it. If they say it's time to go, then that's it. So when Dr. Levy started talking about listing me, I was still working. I was still riding my bike, still totally able bodied. And both me and my partner at the time, we were like, "It doesn't feel like I'm sick enough." But I went on the list anyways.

And we had a bit of trouble finding a blood match, which I thought I had common blood type, A positive. But it's actually not that common. I had an antibody that was also a hard match. So it took us a bit longer. And in that 19 months I had one dry run, which was where they prep you for surgery and then the organs aren't viable, so they send you home. So that was devastating.

But in that 19 months I started getting very sick. And I went on oxygen, and my lungs kept getting infected. And I had septic infection that almost killed me. So my doctor, being able to foresee all of those complications, or that actually you're sick enough that if you start to go down, you'll go down very quickly.

Because I had been sick for so long and my heart was in and out of heart failure, and my insides were quite sick. But I'd never looked sick. So I would say, "If transplant is something that is right for you, then do it even if you don't feel ready for it." And if you don't feel sick enough, because even with the survivor's guilt stuff, where I was like, "But there's so many people that are sicker," or, "Am I even sick enough for this? Is this the right time?" The problem with PH is that you do go down fast. And you don't recover.

For me, my journey was a little bit confusing because for the first five years I was very much in denial, or I never wanted to make a big deal about anything, or make anybody else uncomfortable with me being sick. So I would say it in a way where I just had this little lung disease, or I had this little thing, and I never really felt like I was ever sick enough.

The thing is, is somebody will always be sicker than you. You know. And it took just within the last month or two that I got moved into a place where I felt that actually I was sick enough, and I did deserve these lungs. And that it was okay that I got them. And yeah, people are sicker than you, but that's life, you know. And you're always going to be faced with that.

I think the survivor's guilt part was that I had friends that are at end stage, and they are too sick to get transplant. And they'd be diagnosed longer than me. And they've had a harder journey than me. And I'm over here, riding my bike, and walking my dog and living my life. And really feeling quite removed from the sick girl that I was last year. Because life is so different now. And I guess I feel guilty in that, at times.

When I got home from the hospital, and my partner said something about when I had PH. And it hit both of us, like, "Oh my gosh." I'd been sick for 13 years, and that was my identity, and I was very active in the community, and helped co-running a support group, and be in contact with anybody getting diagnosed. I’d go to the hospital and talk to them, and give them my contact. Being active and of service.

I think that when I got out of the hospital, and I didn't have PH and a little bit of my online community, it felt more quiet. I had a bit of an identity crisis, where I'm like, "Who am I?" I'd been sick for so long, and then I was on the transplant list for what felt like for so long, that that became who I was. And then now I'm this person post-both of those things. And I think as long as I continue to be of service, and to be a contact, and to love and nurture my community, as well as share my blessings that I'm experiencing. Because that is hope. Even though that feels uncomfortable sometimes, I think that that's who I am in the community now. So I'm Natalie, who's had a transplant, and used to have PH.

Transplants been like such an obvious blessing. But it's also been like a really big challenge from being listed and getting really sick really quick near the end there. And then getting, just the whole thing. I would say, yeah, make sure you reach out online. If you're feeling isolated and overwhelmed because there was dark times. There was times when I couldn't get out of bed because I was so crippled with anxiety around dying. And the actual fear around the actual surgery, which is really strange. And I definitely learned some deeper things about myself, and was able to process that through Generation Young Hope, through the other PHers online.

Canadian Natalie Roy is a former pulmonary hypertension patient who received a double-lung transplant in September 2017. She discusses having an identity crisis post PH.

My name is Natalie Roy. I live in Vancouver, British Columbia, Canada. I was diagnosed with, what was called at the time, primary pulmonary hypertension in 2005 when I was 20. Currently, I am one year past new lungs. I just celebrated my one-year lungiversary.

I just started fainting. I started noticing that I was having a hard time walking up hills. At the time, I just wanted to avoid it. I think I was scared, but I was like, "Oh, I'm just dehydrated, or I need a nap, or maybe I haven't eaten enough today." I just was in denial and altered my life patterns to better fit not walking up hills or walking upstairs or whatever.

I fainted walking up a set of stairs. And somebody had called an ambulance. I guess my lips had gone blue, and they couldn't wake me up for over 10 minutes. So I got diagnosed in the emergency room with six months to live, at the time without treatment.

I got on some oral treatment, and made a life change, and moved to Alberta and went to school for chef's school. I did really well for the first five years on just straight oral treatment. And then I went to Australia for a year. And when I came back, I started fainting again. So they put me on a secondary oral. That lasted about two years, and then I started having symptoms and went on IV medication for five years. And got listed for a double-lung transplant November 1st, 2015. And received my lungs September 7th, 2017.

I had always told myself when my team tells me it's time, it doesn't matter if I feel ready or not, then I'll just do it. If they say it's time to go, then that's it. So when Dr. Levy started talking about listing me, I was still working. I was still riding my bike, still totally able bodied. And both me and my partner at the time, we were like, "It doesn't feel like I'm sick enough." But I went on the list anyways.

And we had a bit of trouble finding a blood match, which I thought I had common blood type, A positive. But it's actually not that common. I had an antibody that was also a hard match. So it took us a bit longer. And in that 19 months I had one dry run, which was where they prep you for surgery and then the organs aren't viable, so they send you home. So that was devastating.

But in that 19 months I started getting very sick. And I went on oxygen, and my lungs kept getting infected. And I had septic infection that almost killed me. So my doctor, being able to foresee all of those complications, or that actually you're sick enough that if you start to go down, you'll go down very quickly.

Because I had been sick for so long and my heart was in and out of heart failure, and my insides were quite sick. But I'd never looked sick. So I would say, "If transplant is something that is right for you, then do it even if you don't feel ready for it." And if you don't feel sick enough, because even with the survivor's guilt stuff, where I was like, "But there's so many people that are sicker," or, "Am I even sick enough for this? Is this the right time?" The problem with PH is that you do go down fast. And you don't recover.

For me, my journey was a little bit confusing because for the first five years I was very much in denial, or I never wanted to make a big deal about anything, or make anybody else uncomfortable with me being sick. So I would say it in a way where I just had this little lung disease, or I had this little thing, and I never really felt like I was ever sick enough.

The thing is, is somebody will always be sicker than you. You know. And it took just within the last month or two that I got moved into a place where I felt that actually I was sick enough, and I did deserve these lungs. And that it was okay that I got them. And yeah, people are sicker than you, but that's life, you know. And you're always going to be faced with that.

I think the survivor's guilt part was that I had friends that are at end stage, and they are too sick to get transplant. And they'd be diagnosed longer than me. And they've had a harder journey than me. And I'm over here, riding my bike, and walking my dog and living my life. And really feeling quite removed from the sick girl that I was last year. Because life is so different now. And I guess I feel guilty in that, at times.

When I got home from the hospital, and my partner said something about when I had PH. And it hit both of us, like, "Oh my gosh." I'd been sick for 13 years, and that was my identity, and I was very active in the community, and helped co-running a support group, and be in contact with anybody getting diagnosed. I’d go to the hospital and talk to them, and give them my contact. Being active and of service.

I think that when I got out of the hospital, and I didn't have PH and a little bit of my online community, it felt more quiet. I had a bit of an identity crisis, where I'm like, "Who am I?" I'd been sick for so long, and then I was on the transplant list for what felt like for so long, that that became who I was. And then now I'm this person post-both of those things. And I think as long as I continue to be of service, and to be a contact, and to love and nurture my community, as well as share my blessings that I'm experiencing. Because that is hope. Even though that feels uncomfortable sometimes, I think that that's who I am in the community now. So I'm Natalie, who's had a transplant, and used to have PH.

Transplants been like such an obvious blessing. But it's also been like a really big challenge from being listed and getting really sick really quick near the end there. And then getting, just the whole thing. I would say, yeah, make sure you reach out online. If you're feeling isolated and overwhelmed because there was dark times. There was times when I couldn't get out of bed because I was so crippled with anxiety around dying. And the actual fear around the actual surgery, which is really strange. And I definitely learned some deeper things about myself, and was able to process that through Generation Young Hope, through the other PHers online.

My name's Brooke Paulin. I am from Mississauga, Ontario, just outside of Toronto. I was diagnosed in February of 2014, so I guess four and a half years ago, just shy of five.

I was a competitive athlete growing up. I represented Canada in figure skating, I was a pair skater. So obviously aerobic capacity was pretty much second to none. I "retired", when I was about 21, and it was actually at that point, I'd given up my relationship with the gym, because having been forced to go to the gym my entire life, once I was no longer forced, that was kind of that. But shortly after, I decided to do a charity bike ride with my mother to raise money for palliative care at a local hospital where we live for palliative care. And I just couldn't keep up for the life of me. Biking was ridiculously hard. And I still didn't really think anything of it. I thought I was just out of shape, and this is what not going to the gym does to somebody. And it took about six months for me to realize nope, something is seriously wrong. This is not normal, I should not be short of breath. Granted winters are cold in Toronto, but I should not be short of breath walking across a parking lot in the middle of winter. Not to the extent I was.

I actually ended up going to a walk in clinic, thinking I was just going to have pneumonia, they were going to diagnose me with some sort of maybe viral infection, something affecting my lungs. [That I would] take some sort of medication and I'd be better. Sure enough, I was actually sent to another local hospital, and admitted there to cardiology for 10 days. I have a lucky story, it only took 10 days for me to get the proper diagnosis. I know, unheard of. But that was that. 10 days in cardiology, a referral to our local pulmonary hypertension clinic, which happened a week after diagnosis. And that was where it was confirmed.

During my stay in the hospital in cardiology, it was really just like a fog. I remember being there, I remember medical jargon being thrown around, but I don't remember them ever saying pulmonary hypertension. I know that they did, because both my parents heard it there for the first time. But I have no recollection of that. I did find out actually during that stay, I also have a heart defect. I have an ASD, so that I've obviously had my entire life. But that I recall from that conversation. And then again, I remember a week later going to the clinic at Toronto General hospital, and that's when I remember hearing pulmonary hypertension for the first time, and really understanding, (well maybe not understanding, because I don't think you can understand much in that moment), but hearing it and absorbing it, and honestly, I felt like I was hit with a Mack truck. I didn't understand how two years prior, I was super healthy and super active, and could run for an hour straight and be fine, and then two years later couldn't walk across a parking lot. Literally, the best way I can say it is I felt like I was hit by a Mack truck at the time.

Fortunately, I've always been that kind of person where I want to be busy 24/7. I don't do well with down time, and I think that's actually been my saving grace. Post diagnosis, I jumped right back into work. My boss at the time was super understanding. He's still a mentor. I no longer work for him, but he is a still a mentor of mine in my professional life, and now personal life, obviously as well. But he was super understanding, made allowances for me in the sense that, not only is it physically challenging, it's emotionally challenging, and especially in those few months following diagnosis, there were days where it was just emotional, and sometimes you can’t hide that.

I did my best, but he made allowances for me to take the time when I needed the time. And I actually think that was the best blessing, the best gift anybody could've given me, because that allowed me to live with the diagnosis, figure out what this new normal was going to look like, but continue to work. Which again, for me was what I needed, because keeping busy is I think what helped me actually move into that new normal, into the understanding, into the acceptance almost, and then figuring out this is the hand of cards that I was dealt. How do I now move forward and make sure that I am still living a fulfilling life while taking care of myself and this disease, and doing what I can to raise awareness. Again, I've been super fortunate. I've been pretty stable for the last four and a half years. I'm on a dual therapy, but I know people aren't that lucky. And so what can I do as well, that helps others get the same access and same advantages that I've been given since diagnosis?

My parents, bless the hearts, have jumped in right alongside of me. My mother is actually a huge advocate in kind of the political side. She goes to Queen's Park, which is really the political headquarters in Toronto. She regularly attends open sessions. Now, because of the advocacy work she’s done, she's also been invited to advisory committees. We're trying to do the national pharma care in Canada, and she's actually been invited to speak and kind of share her thoughts, which is amazing. My dad, as well as my mom, they host a yearly golf tournament in Brampton, so again, just outside of Toronto, where we kind of try to get people together. And whether you golf or you don't, come out for the day and have a great time. Let's raise money and awareness. Next year will actually be our fifth year doing that.

And then just little things as well. We all drive with our bumper sticker on the back of our car. We all wear our pulmonary hypertension bracelet, our purple bracelets, on a daily basis. Like I said earlier, I no longer work for the boss that was super understanding post diagnosis. I work for someone else now. I work for a different company now, and they don't know about my pulmonary hypertension yet. That has been my decision, that has been my choice. But previous to that, all of my employers have known. It's doing what we can to try to get our voice out there. And thankfully my parents are much more vocal about it than I am. But I think we make a good team.

I think recognizing again, we hear it a lot, but that your life is going to be different, but it's going to be a new normal. You're going to acclimate to whatever that normal is, and it may not look like what your life looked like two years prior, but it doesn't mean that it still can't be fulfilling, in whatever capacity that is. So for me, that was to make sure that I could still work and stay busy. So not necessarily in a I'm making money kind of way, but whether it's volunteering, whether it's some hobby, if it's knitting. I think keeping yourself busy, that helps your mental health, so just stay busy.

Stay talking. What I did, and hindsight 20/20 I wish I didn't, I pushed some of my friends away. And in those early months, you're trying to understand and wrap your head around this new normal, and this disease that you've never heard of. Now how do you bring your friends in to that too? Into your lowest moment, how do you bring your friends in, and ask them to understand when you're not understanding? Hindsight 20/20, that's the worst thing I could have done. Thankfully, they have all been very understanding, but hindsight 20/20, don't push people away.

That's another big thing, is it's easy to kind of isolate yourself, and think that nobody'll get it, and you're along in this and nobody'll understand. You'd be absolutely wrong. Let people in. And not necessarily into the depth of what you're thinking, especially in those moments, but keep them in your circle, because at the end of the day, that support system is what's going to move you forward, as well.

My name's Brooke Paulin. I am from Mississauga, Ontario, just outside of Toronto. I was diagnosed in February of 2014, so I guess four and a half years ago, just shy of five.

I was a competitive athlete growing up. I represented Canada in figure skating, I was a pair skater. So obviously aerobic capacity was pretty much second to none. I "retired", when I was about 21, and it was actually at that point, I'd given up my relationship with the gym, because having been forced to go to the gym my entire life, once I was no longer forced, that was kind of that. But shortly after, I decided to do a charity bike ride with my mother to raise money for palliative care at a local hospital where we live for palliative care. And I just couldn't keep up for the life of me. Biking was ridiculously hard. And I still didn't really think anything of it. I thought I was just out of shape, and this is what not going to the gym does to somebody. And it took about six months for me to realize nope, something is seriously wrong. This is not normal, I should not be short of breath. Granted winters are cold in Toronto, but I should not be short of breath walking across a parking lot in the middle of winter. Not to the extent I was.

I actually ended up going to a walk in clinic, thinking I was just going to have pneumonia, they were going to diagnose me with some sort of maybe viral infection, something affecting my lungs. [That I would] take some sort of medication and I'd be better. Sure enough, I was actually sent to another local hospital, and admitted there to cardiology for 10 days. I have a lucky story, it only took 10 days for me to get the proper diagnosis. I know, unheard of. But that was that. 10 days in cardiology, a referral to our local pulmonary hypertension clinic, which happened a week after diagnosis. And that was where it was confirmed.

During my stay in the hospital in cardiology, it was really just like a fog. I remember being there, I remember medical jargon being thrown around, but I don't remember them ever saying pulmonary hypertension. I know that they did, because both my parents heard it there for the first time. But I have no recollection of that. I did find out actually during that stay, I also have a heart defect. I have an ASD, so that I've obviously had my entire life. But that I recall from that conversation. And then again, I remember a week later going to the clinic at Toronto General hospital, and that's when I remember hearing pulmonary hypertension for the first time, and really understanding, (well maybe not understanding, because I don't think you can understand much in that moment), but hearing it and absorbing it, and honestly, I felt like I was hit with a Mack truck. I didn't understand how two years prior, I was super healthy and super active, and could run for an hour straight and be fine, and then two years later couldn't walk across a parking lot. Literally, the best way I can say it is I felt like I was hit by a Mack truck at the time.

Fortunately, I've always been that kind of person where I want to be busy 24/7. I don't do well with down time, and I think that's actually been my saving grace. Post diagnosis, I jumped right back into work. My boss at the time was super understanding. He's still a mentor. I no longer work for him, but he is a still a mentor of mine in my professional life, and now personal life, obviously as well. But he was super understanding, made allowances for me in the sense that, not only is it physically challenging, it's emotionally challenging, and especially in those few months following diagnosis, there were days where it was just emotional, and sometimes you can’t hide that.

I did my best, but he made allowances for me to take the time when I needed the time. And I actually think that was the best blessing, the best gift anybody could've given me, because that allowed me to live with the diagnosis, figure out what this new normal was going to look like, but continue to work. Which again, for me was what I needed, because keeping busy is I think what helped me actually move into that new normal, into the understanding, into the acceptance almost, and then figuring out this is the hand of cards that I was dealt. How do I now move forward and make sure that I am still living a fulfilling life while taking care of myself and this disease, and doing what I can to raise awareness. Again, I've been super fortunate. I've been pretty stable for the last four and a half years. I'm on a dual therapy, but I know people aren't that lucky. And so what can I do as well, that helps others get the same access and same advantages that I've been given since diagnosis?

My parents, bless the hearts, have jumped in right alongside of me. My mother is actually a huge advocate in kind of the political side. She goes to Queen's Park, which is really the political headquarters in Toronto. She regularly attends open sessions. Now, because of the advocacy work she’s done, she's also been invited to advisory committees. We're trying to do the national pharma care in Canada, and she's actually been invited to speak and kind of share her thoughts, which is amazing. My dad, as well as my mom, they host a yearly golf tournament in Brampton, so again, just outside of Toronto, where we kind of try to get people together. And whether you golf or you don't, come out for the day and have a great time. Let's raise money and awareness. Next year will actually be our fifth year doing that.

And then just little things as well. We all drive with our bumper sticker on the back of our car. We all wear our pulmonary hypertension bracelet, our purple bracelets, on a daily basis. Like I said earlier, I no longer work for the boss that was super understanding post diagnosis. I work for someone else now. I work for a different company now, and they don't know about my pulmonary hypertension yet. That has been my decision, that has been my choice. But previous to that, all of my employers have known. It's doing what we can to try to get our voice out there. And thankfully my parents are much more vocal about it than I am. But I think we make a good team.

I think recognizing again, we hear it a lot, but that your life is going to be different, but it's going to be a new normal. You're going to acclimate to whatever that normal is, and it may not look like what your life looked like two years prior, but it doesn't mean that it still can't be fulfilling, in whatever capacity that is. So for me, that was to make sure that I could still work and stay busy. So not necessarily in a I'm making money kind of way, but whether it's volunteering, whether it's some hobby, if it's knitting. I think keeping yourself busy, that helps your mental health, so just stay busy.

Stay talking. What I did, and hindsight 20/20 I wish I didn't, I pushed some of my friends away. And in those early months, you're trying to understand and wrap your head around this new normal, and this disease that you've never heard of. Now how do you bring your friends in to that too? Into your lowest moment, how do you bring your friends in, and ask them to understand when you're not understanding? Hindsight 20/20, that's the worst thing I could have done. Thankfully, they have all been very understanding, but hindsight 20/20, don't push people away.

That's another big thing, is it's easy to kind of isolate yourself, and think that nobody'll get it, and you're along in this and nobody'll understand. You'd be absolutely wrong. Let people in. And not necessarily into the depth of what you're thinking, especially in those moments, but keep them in your circle, because at the end of the day, that support system is what's going to move you forward, as well.

My name is Iain Armstrong. I'm chair of PHA UK. I do two jobs, I do that part-time. I'm also consultant nurse in one of the large PH centers in the UK.

I've been working in PH now for 20 years. 20 years ago we thought it would be a good idea if some of the patients I had could be put in with other patients by telephone, and from that grew PHA UK, and next year is our 20th anniversary and we've got four and a half thousand members, which is half of the PH population in the UK. It's grown from a good idea into this big thing.

I was involved in PH when we first saw the first oral medicine, bosentan. And that generated (from when I was working the 18 month prior to that), we could diagnose PH, but there was very little else we could do. We could prepare some people for transplant and that was about it.

And then bosentan came along - and there was flolan at the time, but it was very limited. But what we had then was a licensed therapy. So what was really useful was a collaboration with a pharmaceutical company, in which we had a common agenda. With any support group, any organization like that, you need initial support. You can't create from nothing without that mutual support. Then we became bigger stronger. The community has become bigger because of collaborative work, both nationally and internationally. I think that's a really important aspect of PH community work. It normalizes something that makes people feel very alone. The rarity even in the UK, we've got 7000 patients in the UK. That makes it very rare. You feel very isolated because there's geographics and things like that.

What we're doing this year, we've actually tried to be very specific about our PHawareness Week [October 22-28]. So this year what we're talking about is individuals being about to talk with friends and family about PH. The work that we've done over the last few years - even in the close knit loved family unit, it's actually quite misunderstood when people feel quite isolated. Actually, being able to have a narrative story that you can tell and share is very powerful. We're trying to provide materials in which that can happen, but one of the taglines that we are going to use this year is the place the potentially transplant may have PH. Not because it's the common end pathway, but it generates a response from the listener, saying, "I didn't realize it was that serious." The diagnosis of PH, at first doesn't sound serious at all. If we call it “cancer,” there'd be a natural sort of human response. PH doesn't sound like that, so we've been exploring, listening to our members. So we’re going to use the place of transplantation so we can get some media publicity to grab people's attention onto a story which really can be quite inspirational and helps people to understand in a way that is fresh.

I was doing a PhD in pulmonary hypertension, looking at the place of hope in life threatening illness. I was aware at that time, that it was actually very difficult for the individuals to have a narrative with clinicians about how does this impact on my quality of life? The quality of life tools available were often quite complex, not sensitive enough to look at PH itself. EmPHasis10 is what we call disease specific quality of life measure, and one that could easily be completed frequently in clinic. So when there’s intervention or when there is change. There's ten questions. Two and a half years in development, and it's been around now for nearly five years and it's been translated into the 35th language internationally. What we were looking at, at the time, primarily was a tool, in which an individual could have a conversation with healthcare professionals and say, "This is what it's like." So it's called a patient reported outcome measure. We feel it, and I professionally feel, it’s absolutely crucial. It's a descriptor rather than a predictor of the future. To see that type of tool being used in the management and care of PH, I feel is professionally really important.

And what it does also is it raises the individual patient's health and illness, [and puts how they] experience illness on the agenda. Without any doubt a rare disease in healthcare, a patient struggles and has to fight above his belt. So what we're really keen on doing with our patient advocacy group is that we get an equal slice of healthcare provision. The rarity should not be a punishment. Keep it on the agenda with people, policy makers, healthcare policy makers, etc. It's an expensive disease to treat. It's always been in the UK, what we would call, a political disease. So advocacy with government, healthcare etc. So it's absolutely crucial.

I will say, our success has always been about collaboration with professionals, policy makers, internationally, and pharma. And you can't be successful unless there's collaboration.

My name is Iain Armstrong. I'm chair of PHA UK. I do two jobs, I do that part-time. I'm also consultant nurse in one of the large PH centers in the UK.

I've been working in PH now for 20 years. 20 years ago we thought it would be a good idea if some of the patients I had could be put in with other patients by telephone, and from that grew PHA UK, and next year is our 20th anniversary and we've got four and a half thousand members, which is half of the PH population in the UK. It's grown from a good idea into this big thing.

I was involved in PH when we first saw the first oral medicine, bosentan. And that generated (from when I was working the 18 month prior to that), we could diagnose PH, but there was very little else we could do. We could prepare some people for transplant and that was about it.

And then bosentan came along - and there was flolan at the time, but it was very limited. But what we had then was a licensed therapy. So what was really useful was a collaboration with a pharmaceutical company, in which we had a common agenda. With any support group, any organization like that, you need initial support. You can't create from nothing without that mutual support. Then we became bigger stronger. The community has become bigger because of collaborative work, both nationally and internationally. I think that's a really important aspect of PH community work. It normalizes something that makes people feel very alone. The rarity even in the UK, we've got 7000 patients in the UK. That makes it very rare. You feel very isolated because there's geographics and things like that.

What we're doing this year, we've actually tried to be very specific about our PHawareness Week [October 22-28]. So this year what we're talking about is individuals being about to talk with friends and family about PH. The work that we've done over the last few years - even in the close knit loved family unit, it's actually quite misunderstood when people feel quite isolated. Actually, being able to have a narrative story that you can tell and share is very powerful. We're trying to provide materials in which that can happen, but one of the taglines that we are going to use this year is the place the potentially transplant may have PH. Not because it's the common end pathway, but it generates a response from the listener, saying, "I didn't realize it was that serious." The diagnosis of PH, at first doesn't sound serious at all. If we call it “cancer,” there'd be a natural sort of human response. PH doesn't sound like that, so we've been exploring, listening to our members. So we’re going to use the place of transplantation so we can get some media publicity to grab people's attention onto a story which really can be quite inspirational and helps people to understand in a way that is fresh.

I was doing a PhD in pulmonary hypertension, looking at the place of hope in life threatening illness. I was aware at that time, that it was actually very difficult for the individuals to have a narrative with clinicians about how does this impact on my quality of life? The quality of life tools available were often quite complex, not sensitive enough to look at PH itself. EmPHasis10 is what we call disease specific quality of life measure, and one that could easily be completed frequently in clinic. So when there’s intervention or when there is change. There's ten questions. Two and a half years in development, and it's been around now for nearly five years and it's been translated into the 35th language internationally. What we were looking at, at the time, primarily was a tool, in which an individual could have a conversation with healthcare professionals and say, "This is what it's like." So it's called a patient reported outcome measure. We feel it, and I professionally feel, it’s absolutely crucial. It's a descriptor rather than a predictor of the future. To see that type of tool being used in the management and care of PH, I feel is professionally really important.

And what it does also is it raises the individual patient's health and illness, [and puts how they] experience illness on the agenda. Without any doubt a rare disease in healthcare, a patient struggles and has to fight above his belt. So what we're really keen on doing with our patient advocacy group is that we get an equal slice of healthcare provision. The rarity should not be a punishment. Keep it on the agenda with people, policy makers, healthcare policy makers, etc. It's an expensive disease to treat. It's always been in the UK, what we would call, a political disease. So advocacy with government, healthcare etc. So it's absolutely crucial.

I will say, our success has always been about collaboration with professionals, policy makers, internationally, and pharma. And you can't be successful unless there's collaboration.

Canadian Pulmonary Hypertension Caregiver Darren Dempsey discusses the impact rare disease has had on his family and how stress impacts the entire family.

My name is Darren Dempsey. I am a caregiver. My wife, Nicole, was diagnosed with PH five years ago. It's really affected our family more than I could ever imagine. We've had to deal a change in employment. Change in finances. Change in the amount of work that I have to do around the house. But we have managed pretty well in the last five years.

Definitely was a shock. I knew that it was life altering as soon as they addressed it and they let us know this is pretty serious. My wife was in severe heart failure. We spend, I believe, 11 days in the hospital from time of diagnosis. We basically walking into a clinic, had some testing done, and didn't get home until three days before Christmas. I knew at that point that our life together was going to be very different than what had imagined or what we had anticipated prior to that.

Initially, we had friends over watching the kids and then we had to call mother to ask her to come over and watch the kids. Really trying to verbalize or let them know, "Hey, you got to get to my house and watch my kids because we're not going home anytime soon and we'll address the children when we get home." And when we did, they were babies. They're now eight and ten, but at five years ago it was a different story. They were pretty small and we took our time addressing it. But, you know, their mom was on 24-hour oxygen. When we got home so they knew something was up for sure.

It can be stressful. You know when you've got to spend 40 hours a week doing a full-time job and then coming home and clean up and cut the grass. We have three dogs, that's a lot of work as well. So I definitely know that the toll on my health is, you know, mentally and physically is there. Like I feel it. I know my blood pressure has changed in the last five years. I know that mentally I've become more and more exhausted think about just how I'm going to help my wife more and more. How I can try to keep myself healthy.

There's a lot of work that's involved when one person doesn't have the energy to give as much. And they give a lot in other areas, definitely. It can be stressful, for sure. I know that, like I said, you're stressed, you're worried about future health for your spouse who has PH and it places a toll on you mentally, for sure.

The one piece of advice that I could give a caregiver is this: It's extremely shocking initially and speak to others. Get as much support as you can and know that in a year or two years with good treatment, good doctors, having the right healthcare available, things will get a lot better. It will definitely get a lot better. We are definitely in a much better situation now than we were five years ago. So it does get a lot better.

Canadian Pulmonary Hypertension Caregiver Darren Dempsey discusses the impact rare disease has had on his family and how stress impacts the entire family.

My name is Darren Dempsey. I am a caregiver. My wife, Nicole, was diagnosed with PH five years ago. It's really affected our family more than I could ever imagine. We've had to deal a change in employment. Change in finances. Change in the amount of work that I have to do around the house. But we have managed pretty well in the last five years.

Definitely was a shock. I knew that it was life altering as soon as they addressed it and they let us know this is pretty serious. My wife was in severe heart failure. We spend, I believe, 11 days in the hospital from time of diagnosis. We basically walking into a clinic, had some testing done, and didn't get home until three days before Christmas. I knew at that point that our life together was going to be very different than what had imagined or what we had anticipated prior to that.

Initially, we had friends over watching the kids and then we had to call mother to ask her to come over and watch the kids. Really trying to verbalize or let them know, "Hey, you got to get to my house and watch my kids because we're not going home anytime soon and we'll address the children when we get home." And when we did, they were babies. They're now eight and ten, but at five years ago it was a different story. They were pretty small and we took our time addressing it. But, you know, their mom was on 24-hour oxygen. When we got home so they knew something was up for sure.

It can be stressful. You know when you've got to spend 40 hours a week doing a full-time job and then coming home and clean up and cut the grass. We have three dogs, that's a lot of work as well. So I definitely know that the toll on my health is, you know, mentally and physically is there. Like I feel it. I know my blood pressure has changed in the last five years. I know that mentally I've become more and more exhausted think about just how I'm going to help my wife more and more. How I can try to keep myself healthy.

There's a lot of work that's involved when one person doesn't have the energy to give as much. And they give a lot in other areas, definitely. It can be stressful, for sure. I know that, like I said, you're stressed, you're worried about future health for your spouse who has PH and it places a toll on you mentally, for sure.

The one piece of advice that I could give a caregiver is this: It's extremely shocking initially and speak to others. Get as much support as you can and know that in a year or two years with good treatment, good doctors, having the right healthcare available, things will get a lot better. It will definitely get a lot better. We are definitely in a much better situation now than we were five years ago. So it does get a lot better.

Canadian Pulmonary Hypertension Patient, Marion Roth discusses the importance of clinical trials, fearlessly tackling her bucket list and educating the world about #phaware-ness.

My name is Marion Roth. I'm a 63 year old lady from Milverton, Ontario. I was diagnosed seven and a half years ago. I'm alive, kicking, and looking for more adventure.

I began to believe that I was overweight. I was out of shape, because as I went for my walks daily with my neighbor who is 20 years younger than me. She was going up hills and walking faster than I could. My husband mentioned one night as I was getting ready for bed, you sound like a freight train. I couldn't breathe. I was just about hyperventilating. So, I went to the doctor, and thankfully my doctor was one of the older doctors that sent me to an internist that had worked in an office beside Dr. Sanjay Mehta. I was diagnosed with PH within four weeks.

The immediate plan of action once they had picked it up on the echocardiogram. Then they had to confirm it with the right heart catheterization. From there then we started onto Lasix, tracleer (bosentan) within six months they added adcirca (tadalafil) to it. Then we stabilized with that for a little bit. Then I went into clinical research trial for Nitric oxide. I was testing the machine that carries it. That was a very satisfying thing to have done, because I met a lady a year ago that because of some of the trialing that I did, she now has a very compact little machine for her Nitric oxide that sits over top of her oxygen tank and she has a quality of life.

Quite frankly, if sick people with PH don't do the clinical trials, how do you find it? You can't test healthy people for this stuff. Right? So we have to. Somebody has to take the plunge and do the trials. So, Dr. Mehta asks when you're at a level, whether you're so far down that he thinks this extra drug can help you. Or you're so good that he thinks you can take the challenge on of another, he asks you if you're interested. And, I've said right from the beginning, if there's anything I can do to advance the research or help other people, because clearly I'm going down the tubes. Right? Once you've got it, you're going down. So, you figure if you can help anybody else, let's go for it.

I've got no fear. I was handed a life sentence of three to five years. I beat it. I'm seven and a half years. I'm on borrowed time. I was born into the world on borrowed time. If I can do any good, help somebody else out, that's my mantra. My family is a giving family. We're community oriented. So, why not get tied up in the medical community as well?

I have a daughter that was through Air Cadets. I have a daughter that went through the Girl Guide Program, and it's all community service work. My husband was a Mason and a Shriner. I worked through them with that. I made all the uniforms for his Shriner's' band.

I got the diagnosis on my own. Then when we went to confirm the diagnosis, he came with me. He was devastated, because we're a very tight knit family. We're very close. We do everything together. Once I got him to understand that I was comfortable with it. I would do what I could to fight. I'm not going to sit down and rust out. I'm going down swinging. And, I told him that I would fight it all I could. He still has difficulty with it. So, we go up in peaks and valleys. And when you get down into that valley sometimes, you're not sure you're coming up.

Well, he wants to bubble wrap me. “You can't do this. Don't do that. I'll do this. I'll do that.” It was very hard on him. Both kids were out of the house. Youngest one is in the military. So, she'd been out of the house since she was 17 and well settled and no problem at all. My oldest daughter is my rock. She will travel anywhere with me. If I go to medical appointments and it's a nasty one, she'll volunteer right off the bat to come with me, because she has lots of time through her work that she can spare. Where my husband's a mechanic, and it's hard to take a mechanic off the floor when there's work booked in and you all of a sudden just up and take sick.

The youngest daughter, she's 32 and is still somewhat in denial. The denial is probably more caused, because she's been out of the picture for so long and away from home. So, when she does come home on a weekend or for a week, she sees mom scooting around and doing everything that mom always did. She doesn't see the aftermath of when she leaves and mom's down for two days and can't do jack squat, because she's exhausted and all the symptoms are back up and flaring again. Because you've pushed. You've done too much. We all work together at it, but we've all come to a consensus. When I need help, I ask for it. If I'm not asking for your help, then I'm okay. I can plod through. I can do it.

You know your own limitations. Every mother, every woman knows how far they can push and how far they can go. And, with PH, you know when you've pushed too far. You know the next day. It bites you. And, it depends on if I want to do something, and it's important to me, I do it. And, I know that I'm going to suffer the consequences, but that's my choice. If somebody forces me to do something, and I'm doing it and I don't really want to, that's when my big girl words come out, and I say, no thank you. And, I don't. Some things are worth pushing for. Other things are not. I was really sick for two and a half months. This late spring into summer. It was touch and go. I was scared. But, we came through it. Come up on the other side of it, and we're bouncing again.

If you can think it, you can dream it, you can do it. Your PH team will help you make that reality. But, you've got to be the advocate. You've got to want to do it. I've done everything I want on my bucket list but one thing. And, I can't do it, and I know I can't. So, I just had to drop it off my bucket list. I wanted to go cliff diving. They can't figure a way through physics that I can go down, go down 15 feet and come back up without exploding my heart. So, we took that off the list. But I've done everything else. Horseback riding, the ocean. I've gone down the slides on the cruise ships.

I've done more stuff than I can ever think of. You can't give in. You can't give in to this. You can't let this illness overtake you. You've got to go. You've got to do. My thing is, I want to help and educate. When I got it, it was the question everybody asks, why me? My answer is why not me?

Canadian Pulmonary Hypertension Patient, Marion Roth discusses the importance of clinical trials, fearlessly tackling her bucket list and educating the world about #phaware-ness.

My name is Marion Roth. I'm a 63 year old lady from Milverton, Ontario. I was diagnosed seven and a half years ago. I'm alive, kicking, and looking for more adventure. I began to believe that I was overweight. I was out of shape, because as I went for my walks daily with my neighbor who is 20 years younger than me. She was going up hills and walking faster than I could. My husband mentioned one night as I was getting ready for bed, you sound like a freight train. I couldn't breathe. I was just about hyperventilating. So, I went to the doctor, and thankfully my doctor was one of the older doctors that sent me to an internist that had worked in an office beside Dr. Sanjay Mehta. I was diagnosed with PH within four weeks. The immediate plan of action once they had picked it up on the echocardiogram. Then they had to confirm it with the right heart catheterization. From there then we started onto Lasix, tracleer (bosentan) within six months they added adcirca (tadalafil) to it. Then we stabilized with that for a little bit. Then I went into clinical research trial for Nitric oxide. I was testing the machine that carries it. That was a very satisfying thing to have done, because I met a lady a year ago that because of some of the trialing that I did, she now has a very compact little machine for her Nitric oxide that sits over top of her oxygen tank and she has a quality of life. Quite frankly, if sick people with PH don't do the clinical trials, how do you find it? You can't test healthy people for this stuff. Right? So we have to. Somebody has to take the plunge and do the trials. So, Dr. Mehta asks when you're at a level, whether you're so far down that he thinks this extra drug can help you. Or you're so good that he thinks you can take the challenge on of another, he asks you if you're interested. And, I've said right from the beginning, if there's anything I can do to advance the research or help other people, because clearly I'm going down the tubes. Right? Once you've got it, you're going down. So, you figure if you can help anybody else, let's go for it. I've got no fear. I was handed a life sentence of three to five years. I beat it. I'm seven and a half years. I'm on borrowed time. I was born into the world on borrowed time. If I can do any good, help somebody else out, that's my mantra. My family is a giving family. We're community oriented. So, why not get tied up in the medical community as well? I have a daughter that was through Air Cadets. I have a daughter that went through the Girl Guide Program, and it's all community service work. My husband was a Mason and a Shriner. I worked through them with that. I made all the uniforms for his Shriner's' band. I got the diagnosis on my own. Then when we went to confirm the diagnosis, he came with me. He was devastated, because we're a very tight knit family. We're very close. We do everything together. Once I got him to understand that I was comfortable with it. I would do what I could to fight. I'm not going to sit down and rust out. I'm going down swinging. And, I told him that I would fight it all I could. He still has difficulty with it. So, we go up in peaks and valleys. And when you get down into that valley sometimes, you're not sure you're coming up. Well, he wants to bubble wrap me. “You can't do this. Don't do that. I'll do this. I'll do that.” It was very hard on him. Both kids were out of the house. Youngest one is in the military. So, she'd been out of the house since she was 17 and well settled and no problem at all. My oldest daughter is my rock. She will travel anywhere with me. If I go to medical appointments and it's a nasty one, she'll volunteer right off the bat to come with me, because she has lots of time through her work that she can spare. Where my husband's a mechanic, and it's hard to take a mechanic off the floor when there's work booked in and you all of a sudden just up and take sick. The youngest daughter, she's 32 and is still somewhat in denial. The denial is probably more caused, because she's been out of the picture for so long and away from home. So, when she does come home on a weekend or for a week, she sees mom scooting around and doing everything that mom always did. She doesn't see the aftermath of when she leaves and mom's down for two days and can't do jack squat, because she's exhausted and all the symptoms are back up and flaring again. Because you've pushed. You've done too much. We all work together at it, but we've all come to a consensus. When I need help, I ask for it. If I'm not asking for your help, then I'm okay. I can plod through. I can do it. You know your own limitations. Every mother, every woman knows how far they can push and how far they can go. And, with PH, you know when you've pushed too far. You know the next day. It bites you. And, it depends on if I want to do something, and it's important to me, I do it. And, I know that I'm going to suffer the consequences, but that's my choice. If somebody forces me to do something, and I'm doing it and I don't really want to, that's when my big girl words come out, and I say, no thank you. And, I don't. Some things are worth pushing for. Other things are not. I was really sick for two and a half months. This late spring into summer. It was touch and go. I was scared. But, we came through it. Come up on the other side of it, and we're bouncing again. If you can think it, you can dream it, you can do it. Your PH team will help you make that reality. But, you've got to be the advocate. You've got to want to do it. I've done everything I want on my bucket list but one thing. And, I can't do it, and I know I can't. So, I just had to drop it off my bucket list. I wanted to go cliff diving. They can't figure a way through physics that I can go down, go down 15 feet and come back up without exploding my heart. So, we took that off the list. But I've done everything else. Horseback riding, the ocean. I've gone down the slides on the cruise ships. I've done more stuff than I can ever think of. You can't give in. You can't give in to this. You can't let this illness overtake you. You've got to go. You've got to do. My thing is, I want to help and educate. When I got it, it was the question everybody asks, why me? My answer is why not me? I'm Marion Roth, and I am aware that I am rare.

Kristen Wieneke is a long term pulmonary hypertension survivor. She was diagnosed in 2003 during open heart surgery to repair an ASD. She discusses the stigma that comes with rare disease, the importance of clinical trials and why patients must be their own advocate.

I'm Kristen Wieneke, and I'm a pulmonary hypertension patient. I was diagnosed with pulmonary hypertension back in 2003 during open heart surgery to repair an ASD. They confirmed the pulmonary hypertension during surgery when I wasn't breathing on my own and had to be left on the ventilator for several more hours. They didn't expect me to live more than five years after that, but here it is almost 15 years later, and I'm still here.

It's been a very emotional roller coaster from the beginning. I was told if I didn't take care of myself and I didn't do everything that the doctors told me I was going to do, I wasn't going to live. So I tried to follow their advice and do what I felt was best for me. Which I didn't always follow the doctor's advice. I had to veer off and learn about my own body and learn about how I responded to the medication and how maybe IV wasn't best for me, because of all of the emotional things that go along with that. To be able to work with a pump that you have attached to you and an open line directly into your heart is not something you want to show everybody. And then when they do see it, and they think, "Wow. What's wrong with you?" And then they're afraid me, afraid of the line, afraid because that looks scary coming right out of your skin.

To get past all of that stigma, those people that didn't understand, which brings more awareness. People have to understand that I have this disease, yes. But I'm still a person. I still have feelings. You still want to be included in events and things, and they don't want to include you because they're afraid you're going to break and you're going to pass out or I'm going to have a heart attack or I'm going to ... I don't know what they thought. But it's very emotional. And then every year that went by, and I was still doing okay. And I tried this medication and I tried that new trial. And I was grateful for every birthday that I had and I was still able to function. And I'm still able to function without being on continuous oxygen. Just at night. So I'm grateful for that.

I had to change my mindset that I'm not going to worry about what other people think. And I have to just focus on myself. And if I feel okay, I will go out and participate. If I don't, then I just tell them, "I'm just going to stay home today." I'm okay with that. I've learned that that's okay to do. I've learned that it's okay to say no. That's my coping, that I felt like I used to have to participate in everything. But when I stopped working, that was probably one of the best things I could have done for myself. I've accepted the disease, and I'm able to just relax. And I can be home now and I don't get as sad, because I know it's just today. Tomorrow might be different. So I can take it day by day. I live day by day. I don't try to think about the future because that's too overwhelming. I don't know what the future brings. I only know what today has. I can only focus on right now.

I wish I would have known that having this disease doesn't mean that I'm going to die. Fifteen years ago when I was diagnosed, it was very serious and there wasn't many treatments for it. We only had a handful, four or five possible drugs that were available. Now we have 14. Back then, it was very grim. And I wish I would have known that it's going to get better. It's not a death sentence. Pulmonary hypertension is no longer a death sentence. There's treatments available. There's support available. There's a number of people that can help you. And I wish I would have been able to find them when I was diagnosed.

You have to be your own advocate. You have to know your body. Do your research on the medications. Just because your doctor says I want to put you on this drug, you have to know why that drug and not this other drug might be better for you. There's support. There's help out there. There's people you can talk to. If you don't get along with your doctor, find a doctor you can talk to. Find somebody that will listen to you. This disease is so individual that every person and every patient is different. So what might be true for one person isn't going to be true for another person. Everybody has to find out what is good for them. And a doctor that listens to you and knows that your body is your body and not theirs to play with I think is very important. I've gone through a few doctors to find somebody that listens.

Clinical trials is extremely important because it helps doctors, it helps the pharmaceutical companies learn about what works and what doesn't work. Without the trials, we wouldn't have new drugs. We wouldn't have the tweaking of the drugs. I encourage all patients to participate in clinical trials. It's rewarding to know that you're participating in something that can help somebody else.

I do a PH walk in Milwaukee. I encourage everybody in the Milwaukee area to come and participate in my event. I also have a support group that I like to help people and advocate for them, as well. I encourage new patients to go to a support group. I didn't have that when I was diagnosed. I actually was laughed at when I was going to my first support group meeting because I was only 30 years old, and there was a woman there who was a lot older than me, probably twice my age who has been living with pulmonary hypertension for 10, maybe 15 years. She was very active. She was on IV drug, used oxygen. And her pulmonary pressures were double what mine were. She looked at me and saw that I just had open heart surgery and said, "Oh, you're going to be fine. My pressures are this and I'm fine. I do all this stuff."

I was 30 years old. A month ago, I was fine. I didn't have this disease. I didn't have anything. I was working full time. I had just bought a house. Now all of a sudden, I have this disease. My whole life is changed. My world was shattered. And she was blowing me off like I was a joke. There was no support at all. It was awful.

I love to see other people in their own elements talking about living with the disease. Essentially, we're all just trying to live with it. And to hear other people that I can relate to is empowering and encouraging to know that there's other people trying to live the same way I am because this disease is so unknown.

Kristen Wieneke is a long term pulmonary hypertension survivor. She was diagnosed in 2003 during open heart surgery to repair an ASD. She discusses the stigma that comes with rare disease, the importance of clinical trials and why patients must be their own advocate.

I'm Kristen Wieneke, and I'm a pulmonary hypertension patient. I was diagnosed with pulmonary hypertension back in 2003 during open heart surgery to repair an ASD. They confirmed the pulmonary hypertension during surgery when I wasn't breathing on my own and had to be left on the ventilator for several more hours. They didn't expect me to live more than five years after that, but here it is almost 15 years later, and I'm still here.

It's been a very emotional roller coaster from the beginning. I was told if I didn't take care of myself and I didn't do everything that the doctors told me I was going to do, I wasn't going to live. So I tried to follow their advice and do what I felt was best for me. Which I didn't always follow the doctor's advice. I had to veer off and learn about my own body and learn about how I responded to the medication and how maybe IV wasn't best for me, because of all of the emotional things that go along with that. To be able to work with a pump that you have attached to you and an open line directly into your heart is not something you want to show everybody. And then when they do see it, and they think, "Wow. What's wrong with you?" And then they're afraid me, afraid of the line, afraid because that looks scary coming right out of your skin.

To get past all of that stigma, those people that didn't understand, which brings more awareness. People have to understand that I have this disease, yes. But I'm still a person. I still have feelings. You still want to be included in events and things, and they don't want to include you because they're afraid you're going to break and you're going to pass out or I'm going to have a heart attack or I'm going to ... I don't know what they thought. But it's very emotional. And then every year that went by, and I was still doing okay. And I tried this medication and I tried that new trial. And I was grateful for every birthday that I had and I was still able to function. And I'm still able to function without being on continuous oxygen. Just at night. So I'm grateful for that.

I had to change my mindset that I'm not going to worry about what other people think. And I have to just focus on myself. And if I feel okay, I will go out and participate. If I don't, then I just tell them, "I'm just going to stay home today." I'm okay with that. I've learned that that's okay to do. I've learned that it's okay to say no. That's my coping, that I felt like I used to have to participate in everything. But when I stopped working, that was probably one of the best things I could have done for myself. I've accepted the disease, and I'm able to just relax. And I can be home now and I don't get as sad, because I know it's just today. Tomorrow might be different. So I can take it day by day. I live day by day. I don't try to think about the future because that's too overwhelming. I don't know what the future brings. I only know what today has. I can only focus on right now.

I wish I would have known that having this disease doesn't mean that I'm going to die. Fifteen years ago when I was diagnosed, it was very serious and there wasn't many treatments for it. We only had a handful, four or five possible drugs that were available. Now we have 14. Back then, it was very grim. And I wish I would have known that it's going to get better. It's not a death sentence. Pulmonary hypertension is no longer a death sentence. There's treatments available. There's support available. There's a number of people that can help you. And I wish I would have been able to find them when I was diagnosed.

You have to be your own advocate. You have to know your body. Do your research on the medications. Just because your doctor says I want to put you on this drug, you have to know why that drug and not this other drug might be better for you. There's support. There's help out there. There's people you can talk to. If you don't get along with your doctor, find a doctor you can talk to. Find somebody that will listen to you. This disease is so individual that every person and every patient is different. So what might be true for one person isn't going to be true for another person. Everybody has to find out what is good for them. And a doctor that listens to you and knows that your body is your body and not theirs to play with I think is very important. I've gone through a few doctors to find somebody that listens.

Clinical trials is extremely important because it helps doctors, it helps the pharmaceutical companies learn about what works and what doesn't work. Without the trials, we wouldn't have new drugs. We wouldn't have the tweaking of the drugs. I encourage all patients to participate in clinical trials. It's rewarding to know that you're participating in something that can help somebody else.

I do a PH walk in Milwaukee. I encourage everybody in the Milwaukee area to come and participate in my event. I also have a support group that I like to help people and advocate for them, as well. I encourage new patients to go to a support group. I didn't have that when I was diagnosed. I actually was laughed at when I was going to my first support group meeting because I was only 30 years old, and there was a woman there who was a lot older than me, probably twice my age who has been living with pulmonary hypertension for 10, maybe 15 years. She was very active. She was on IV drug, used oxygen. And her pulmonary pressures were double what mine were. She looked at me and saw that I just had open heart surgery and said, "Oh, you're going to be fine. My pressures are this and I'm fine. I do all this stuff."

I was 30 years old. A month ago, I was fine. I didn't have this disease. I didn't have anything. I was working full time. I had just bought a house. Now all of a sudden, I have this disease. My whole life is changed. My world was shattered. And she was blowing me off like I was a joke. There was no support at all. It was awful.

I love to see other people in their own elements talking about living with the disease. Essentially, we're all just trying to live with it. And to hear other people that I can relate to is empowering and encouraging to know that there's other people trying to live the same way I am because this disease is so unknown.

]]>fullEpisode 197 - Kristen WienekeKristen Wieneke - Pulmonary HypertensionThu, 11 Oct 2018 12:00:00 +0000Kristen Wieneke is a long term pulmonary hypertension survivor. She was diagnosed in 2003 during open heart surgery to repair an ASD. She discusses the stigma that comes with rare disease, the importance of clinical trials and why patients must be their own advocate.

]]>Kristen Wieneke is a long term pulmonary hypertension survivor. She was diagnosed in 2003 during open heart surgery to repair an ASD. She discusses the stigma that comes with rare disease, the importance of clinical trials and why patients must be their own advocate.

]]>07:06cleanblood,research,heart,pediatric,awareness,asthma,iv,breathless,asd,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdisease,subqKristen Wieneke is a long term pulmonary hypertension survivor. She was diagnosed in 2003 during open heart surgery to repair an ASD. She discusses the stigma that comes with rare disease, the importance of clinical trials and why patients must be their own advocate.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware. Engage for a cure: www.phaware.global/donate #phaware @antidote_me
293fullphaware global associationAbby Sherwood - phaware® interview 196Tue, 09 Oct 2018 12:00:00 +0000

Abby Sherwood discusses navigating life with pulmonary hypertension on the 15th anniversary of her PH diagnosis.

My name is Abby Sherwood, and I am a pulmonary hypertension patient.

From what I've been told, I think I was probably starting to slow down. Two-year-olds, they're very energetic and they like to play, and they like to run around and everything. What probably happened was I probably started slowing down, and my parents probably noticed that I was more out of breath than usual, so they took me to my doctor. He did an echocardiogram and he did a right heart catheterization.

They diagnosed me with PH right off the bat. There wasn't any other thing it could have been. My mom has described how she was in the room, and she said she was so mad she could spit nails. That was probably pretty accurate. Then again, I was really young, so I don't remember. October 2018, it will be 15 years.

I don't remember being healthy, so that's not one of my challenges. One of those things I hear is these adults saying, "Oh yeah, I was diagnosed five years ago." They remember being able to run around, do sports. They have been able to be healthy, and live a healthy life. I don't remember that, so I don't have a great sense of loss for an old life, because I don't really have an old life that I remember.

Early elementary school was kind of difficult because I was on epoprostenol (IV therapy). You have to have ice packs in with your pump. Those had to be changed regularly at school, and I had to go down to the office for medicine during the day. That was kind of difficult.

Later on, sort of in middle school, I started subq, and for anyone that's been on subq, they know that when you start a new site there is excruciating pain wherever it is. Even if I had a site in my arm, I always did it in my left arm, because I'm right handed. If I had it in my right hand, then I'd just have really bad pain and I wouldn't be able to write, and that wouldn't work.

Even if it was in my left arm, it would affect my ability to walk and do things. When you walk, you have a bouncing motion. We had to either put it in a sling, or if I had the site in my stomach, which was also a frequent spot, I would be in a wheelchair at school, which would be really difficult because I had a couple of friends that would be able to wheel me around.

That wasn't working for me because my condition started to decline, and I had to be put on oxygen most of my day, and I had to start taking tanks to school, so that wasn't that fun. I had to drag those around everywhere, and I had to walk everywhere. It was a small school, so it wasn't that hard, but it was still fairly difficult.

Then we went back on the Broviac central line, and it started to get a little better, but I was still on oxygen. That was still difficult. Then I had Potts Shunt surgery in eighth grade, so that I had under oxygenated blood going to my lower extremities, and the better oxygenated blood going up here to my heart, and my lungs, and my brain. That helped me tremendously, but I missed a lot of school.

When I finally got back, I was behind on a bunch of things. We used to do this thing called accelerated reading, or AR, where we had to read books, take tests on them and then we'd get points. I was dreadfully behind on that. My teacher was just like, "It's understandable why you missed that. We're just going to excuse you."

While I was recovering at home, we had my friends and their families sign up on this sheet, and they would bring me food. They would bring meals. My mom could just put those on plates and we could all eat those instead of her having to make stuff, so she could take care of me. That was great because it just made me feel good that my classmates cared about me.

Then I had to leave those people whom I had known for, some of them for those nine years that I had been there. Then I went to this gigantic high school where I knew nobody. I had to get this thing called a 504 plan, where I can leave classes early. If I needed to, I could get an extra set of books, take those home. I really only used it to leave class early.

I always had to worry, I still have to worry about this, I'll explain it to my teachers that I need to leave class early. I'm like, "Hey I've got this thing. I've got to leave early." I don't have to explain it every day. Then I'll get there, and I'll have a sub, and I'll be like oh great. I'll have to explain that to them. They're always usually very good about it, and they'll be like, "Oh, okay sure go."

It gets better honestly because sometimes you're going to struggle with it. You're going to be like, I'm sick, I can't do a lot of things, this kind of sucks. For me, I was a cheerleader. When I started subq, my condition started declining. I couldn't do that anymore. I was really upset about it, but then I realized I could be a secondary coach, so I could hang out with the cheerleaders, do some of the dances, teach some of them the moves. I could be there, but I just couldn't be up on stage doing all the moves. I could help with these kids. That’s what made me really happy.

It's going to suck at times, but it does get better. Do what you can. Let's say you like soccer, or sports, or something active, you can find new things that you like. For me, that's like reading and writing. I can sit and do something calm. You have to find stuff that will fit your illness, and stuff that you can actually do without overdoing it. I think that's part of my problem. I'll do something, and I'll be like, "Oh yeah, I'm fine. It's whatever. It's cool." Then later in that day I'll be like, "Yeah, I'm exhausted." Don't overdo it.

Learn your body. Know when you're doing too much for yourself. As you get older, you might go into high school, and you're going to meet some mean people. They're making fun of something you can't control. That's really low for people. That's not something anyone should be doing. Try not to let it bug you. My mom says, you have to grow duck feathers. Water can represent mean comments from people, or things in your life that are coming at you, trying to bring you down, just get duck feathers and let it roll off.

Abby Sherwood discusses navigating life with pulmonary hypertension on the 15th anniversary of her PH diagnosis.

My name is Abby Sherwood, and I am a pulmonary hypertension patient.

From what I've been told, I think I was probably starting to slow down. Two-year-olds, they're very energetic and they like to play, and they like to run around and everything. What probably happened was I probably started slowing down, and my parents probably noticed that I was more out of breath than usual, so they took me to my doctor. He did an echocardiogram and he did a right heart catheterization.

They diagnosed me with PH right off the bat. There wasn't any other thing it could have been. My mom has described how she was in the room, and she said she was so mad she could spit nails. That was probably pretty accurate. Then again, I was really young, so I don't remember. October 2018, it will be 15 years.

I don't remember being healthy, so that's not one of my challenges. One of those things I hear is these adults saying, "Oh yeah, I was diagnosed five years ago." They remember being able to run around, do sports. They have been able to be healthy, and live a healthy life. I don't remember that, so I don't have a great sense of loss for an old life, because I don't really have an old life that I remember.

Early elementary school was kind of difficult because I was on epoprostenol (IV therapy). You have to have ice packs in with your pump. Those had to be changed regularly at school, and I had to go down to the office for medicine during the day. That was kind of difficult.

Later on, sort of in middle school, I started subq, and for anyone that's been on subq, they know that when you start a new site there is excruciating pain wherever it is. Even if I had a site in my arm, I always did it in my left arm, because I'm right handed. If I had it in my right hand, then I'd just have really bad pain and I wouldn't be able to write, and that wouldn't work.

Even if it was in my left arm, it would affect my ability to walk and do things. When you walk, you have a bouncing motion. We had to either put it in a sling, or if I had the site in my stomach, which was also a frequent spot, I would be in a wheelchair at school, which would be really difficult because I had a couple of friends that would be able to wheel me around.

That wasn't working for me because my condition started to decline, and I had to be put on oxygen most of my day, and I had to start taking tanks to school, so that wasn't that fun. I had to drag those around everywhere, and I had to walk everywhere. It was a small school, so it wasn't that hard, but it was still fairly difficult.

Then we went back on the Broviac central line, and it started to get a little better, but I was still on oxygen. That was still difficult. Then I had Potts Shunt surgery in eighth grade, so that I had under oxygenated blood going to my lower extremities, and the better oxygenated blood going up here to my heart, and my lungs, and my brain. That helped me tremendously, but I missed a lot of school.

When I finally got back, I was behind on a bunch of things. We used to do this thing called accelerated reading, or AR, where we had to read books, take tests on them and then we'd get points. I was dreadfully behind on that. My teacher was just like, "It's understandable why you missed that. We're just going to excuse you."

While I was recovering at home, we had my friends and their families sign up on this sheet, and they would bring me food. They would bring meals. My mom could just put those on plates and we could all eat those instead of her having to make stuff, so she could take care of me. That was great because it just made me feel good that my classmates cared about me.

Then I had to leave those people whom I had known for, some of them for those nine years that I had been there. Then I went to this gigantic high school where I knew nobody. I had to get this thing called a 504 plan, where I can leave classes early. If I needed to, I could get an extra set of books, take those home. I really only used it to leave class early.

I always had to worry, I still have to worry about this, I'll explain it to my teachers that I need to leave class early. I'm like, "Hey I've got this thing. I've got to leave early." I don't have to explain it every day. Then I'll get there, and I'll have a sub, and I'll be like oh great. I'll have to explain that to them. They're always usually very good about it, and they'll be like, "Oh, okay sure go."

It gets better honestly because sometimes you're going to struggle with it. You're going to be like, I'm sick, I can't do a lot of things, this kind of sucks. For me, I was a cheerleader. When I started subq, my condition started declining. I couldn't do that anymore. I was really upset about it, but then I realized I could be a secondary coach, so I could hang out with the cheerleaders, do some of the dances, teach some of them the moves. I could be there, but I just couldn't be up on stage doing all the moves. I could help with these kids. That’s what made me really happy.

It's going to suck at times, but it does get better. Do what you can. Let's say you like soccer, or sports, or something active, you can find new things that you like. For me, that's like reading and writing. I can sit and do something calm. You have to find stuff that will fit your illness, and stuff that you can actually do without overdoing it. I think that's part of my problem. I'll do something, and I'll be like, "Oh yeah, I'm fine. It's whatever. It's cool." Then later in that day I'll be like, "Yeah, I'm exhausted." Don't overdo it.

Learn your body. Know when you're doing too much for yourself. As you get older, you might go into high school, and you're going to meet some mean people. They're making fun of something you can't control. That's really low for people. That's not something anyone should be doing. Try not to let it bug you. My mom says, you have to grow duck feathers. Water can represent mean comments from people, or things in your life that are coming at you, trying to bring you down, just get duck feathers and let it roll off.

]]>06:11cleanblood,research,heart,pediatric,awareness,asthma,iv,breathless,rare,ph,potts,pulmonary,raredisease,shunt,catheterization,pulmonaryhypertension,phaware,lungdisease,subq"I don't remember being healthy, that's not one of my challenges. I don't have a great sense of loss for an old life, because I don't really have an old life that I remember." Abby Sherwood on 15 years with pulmonary hypertension @phaware Ep 196 #phaware #YoungwithPAH
Learn more about pulmonary hypertension trials at www.phaware.global. Follow us @phaware
Engage for a cure: www.phaware.global/donate #phaware #pottsshunt 292fullphaware global associationLaurie WItherwax - phaware® interview 195Fri, 05 Oct 2018 12:00:00 +0000

Laurie Witherwax is the Vice President of Project Management atWorldwide Clinical Trials, which provides drug development services from Early Phase and Bioanalytical Sciences, through Phase II, III and IV. In this episode, Laurie discusses the role of CROs (clinical research organizations). Laurie was the Program Director for global Phase III cardiovascular trials encompassing 38 countries across 620 sites, including 3,400 patients, and other trials involving complex and rare diseases. Her responsibilities have spanned the full duration of studies, from start-up to project close-out and review.

Hi. I'm Laurie Witherwax. I'm Vice President of Project Management at Worldwide Clinical Trials, which is a clinical research organization. My connection to pulmonary hypertension is that I work on PH studies and am very interested in working with our patients, and enjoy working, and learning more about pulmonary hypertension through our patients.

Clinical trials are paramount, and I would say I'm especially interested working through CROs or clinical research organizations because through clinical research organizations, we're able to get the drugs and treatments to the market faster to patients who need them. In fact, based on a Tufts study, we're able to get drugs to market 30 percent faster than if they were not done through a clinical research organization.

A clinical research organization really accelerates the scientific and medical innovation by bringing all of the touch points that happen in clinical research into one organization. For example, CROs are composed of physicians, scientists, and multiple people with therapeutic, data, science, regulatory experience, operations experience, and that's all brought into one organization. Then with that expertise, we're able then to collaborate with academic institutions, physicians out across the globe, and then also work with biotechnology, pharmaceutical companies, and then from there, all of that's brought together under a clinical research organization, where we can then share that information as we continue clinical trials in accumulative fashion.

What makes a clinical research organization different is that we have all of those people with expertise that I mentioned. We got the scientific expertise. We've got the operational expertise, and because we work with a variety of pharmaceutical and biotechnology companies and organizations, we're able to replicate clinical trials, we're able to become more efficient at it, and then we're also to take the knowledge that we learn from those clinical trials such as import/exporting across the globe with devices or drugs. We're able to do that and improve upon it each time.

We act as a hub for clinical research through many touch points. With a clinical trial, we gain a specific therapeutic experience, so we might not just do one PH study. We may do several, and we may do several that are interrelated, and we may do several that use devices or use other modes of delivery, and we're able, again, to learn what patients like, what they don't like, and we're also able to learn how best to execute those studies, so we get them done with greater accuracy. We get them done with more understanding of what the patient needs, and also we develop relationships with the physicians who are participating in these clinical trials.

I think it's important for the patients to understand that we're governed. We're governed by strict regulatory and quality processes that are really designed to focus on patient safety and prioritize patient safety as number one. For example, we have a complex set of documents that we have to get regulatory approval for before we can even begin a clinical trial, and then we also are required to monitor very closely the data that's collected from patients from clinical trials to ensure the safety, and then also we collect all of the data so that it can be analyzed to ensure patient efficacy, as well.

A lot of patients, first of all, will ask, "I'm just going to get a placebo, right?" And it's not always the case. A patient may get a standard of care. Clinical trials do not put the patient's safety in jeopardy at any point, so it's really designed to make sure that we're looking at the standard of care and being able to determine the effect and safety of a new drug versus not having it out on the market. I think, number one, patients just assume that they're just not going to have adequate access to maybe a drug that's working quite well. The other thing is that often times, drugs are provided after their participation in the treatment, so if they are on placebo for any point in time, after the treatment period is up, they do have access to the active drug.

And then number two, I think people just ask, "What's in it for me? And how can I know that I'm advancing the research in my particular condition or disease?" I think what's in it for them is, of course, they get enhanced care. They often will see their physician more often, or they'll certainly be monitored more often. They'll learn more about their care, and then also I think it's good. It gets their caregivers involved often times so that their caregivers understand the disease a little bit better.

I would like to say just at the end of the clinical trial though, I think it's important that patients understand that we pull in all the data, it's analyzed, and everybody looks to have just the expectation, and hope that the patient will actually have access to this drug. It sometimes takes a long time. It's a long effort, and I just want to thank the patients, any patients who participate in clinical trial for doing that. It's important.

Laurie Witherwax is the Vice President of Project Management atWorldwide Clinical Trials, which provides drug development services from Early Phase and Bioanalytical Sciences, through Phase II, III and IV. In this episode, Laurie discusses the role of CROs (clinical research organizations). Laurie was the Program Director for global Phase III cardiovascular trials encompassing 38 countries across 620 sites, including 3,400 patients, and other trials involving complex and rare diseases. Her responsibilities have spanned the full duration of studies, from start-up to project close-out and review.

Hi. I'm Laurie Witherwax. I'm Vice President of Project Management at Worldwide Clinical Trials, which is a clinical research organization. My connection to pulmonary hypertension is that I work on PH studies and am very interested in working with our patients, and enjoy working, and learning more about pulmonary hypertension through our patients.

Clinical trials are paramount, and I would say I'm especially interested working through CROs or clinical research organizations because through clinical research organizations, we're able to get the drugs and treatments to the market faster to patients who need them. In fact, based on a Tufts study, we're able to get drugs to market 30 percent faster than if they were not done through a clinical research organization.

A clinical research organization really accelerates the scientific and medical innovation by bringing all of the touch points that happen in clinical research into one organization. For example, CROs are composed of physicians, scientists, and multiple people with therapeutic, data, science, regulatory experience, operations experience, and that's all brought into one organization. Then with that expertise, we're able then to collaborate with academic institutions, physicians out across the globe, and then also work with biotechnology, pharmaceutical companies, and then from there, all of that's brought together under a clinical research organization, where we can then share that information as we continue clinical trials in accumulative fashion.

What makes a clinical research organization different is that we have all of those people with expertise that I mentioned. We got the scientific expertise. We've got the operational expertise, and because we work with a variety of pharmaceutical and biotechnology companies and organizations, we're able to replicate clinical trials, we're able to become more efficient at it, and then we're also to take the knowledge that we learn from those clinical trials such as import/exporting across the globe with devices or drugs. We're able to do that and improve upon it each time.

We act as a hub for clinical research through many touch points. With a clinical trial, we gain a specific therapeutic experience, so we might not just do one PH study. We may do several, and we may do several that are interrelated, and we may do several that use devices or use other modes of delivery, and we're able, again, to learn what patients like, what they don't like, and we're also able to learn how best to execute those studies, so we get them done with greater accuracy. We get them done with more understanding of what the patient needs, and also we develop relationships with the physicians who are participating in these clinical trials.

I think it's important for the patients to understand that we're governed. We're governed by strict regulatory and quality processes that are really designed to focus on patient safety and prioritize patient safety as number one. For example, we have a complex set of documents that we have to get regulatory approval for before we can even begin a clinical trial, and then we also are required to monitor very closely the data that's collected from patients from clinical trials to ensure the safety, and then also we collect all of the data so that it can be analyzed to ensure patient efficacy, as well.

A lot of patients, first of all, will ask, "I'm just going to get a placebo, right?" And it's not always the case. A patient may get a standard of care. Clinical trials do not put the patient's safety in jeopardy at any point, so it's really designed to make sure that we're looking at the standard of care and being able to determine the effect and safety of a new drug versus not having it out on the market. I think, number one, patients just assume that they're just not going to have adequate access to maybe a drug that's working quite well. The other thing is that often times, drugs are provided after their participation in the treatment, so if they are on placebo for any point in time, after the treatment period is up, they do have access to the active drug.

And then number two, I think people just ask, "What's in it for me? And how can I know that I'm advancing the research in my particular condition or disease?" I think what's in it for them is, of course, they get enhanced care. They often will see their physician more often, or they'll certainly be monitored more often. They'll learn more about their care, and then also I think it's good. It gets their caregivers involved often times so that their caregivers understand the disease a little bit better.

I would like to say just at the end of the clinical trial though, I think it's important that patients understand that we pull in all the data, it's analyzed, and everybody looks to have just the expectation, and hope that the patient will actually have access to this drug. It sometimes takes a long time. It's a long effort, and I just want to thank the patients, any patients who participate in clinical trial for doing that. It's important.

]]>fullEpisode 195 - Laurie WitherwaxLaurie Witherwax - Clinical TrialsThu, 04 Oct 2018 12:00:00 +0000Laurie Witherwax is the Vice President of Project Management atWorldwide Clinical Trials, which provides drug development services from Early Phase and Bioanalytical Sciences, through Phase II, III and IV. In this episode, Laurie discusses the role of CROs (clinical research organizations). Laurie was the Program Director for global Phase III cardiovascular trials encompassing 38 countries across 620 sites, including 3,400 patients, and other trials involving complex and rare diseases. Her responsibilities have spanned the full duration of studies, from start-up to project close-out and review.

]]>Laurie Witherwax is the Vice President of Project Management atWorldwide Clinical Trials, which provides drug development services from Early Phase and Bioanalytical Sciences, through Phase II, III and IV. In this episode, Laurie discusses the role of CROs (clinical research organizations). Laurie was the Program Director for global Phase III cardiovascular trials encompassing 38 countries across 620 sites, including 3,400 patients, and other trials involving complex and rare diseases. Her responsibilities have spanned the full duration of studies, from start-up to project close-out and review.

]]>05:08cleancro,blood,research,heart,awareness,asthma,iv,breathless,rare,ph,pulmonary,raredisease,catheterization,clinicaltrials,pulmonaryhypertension,phaware,lungdiseaseLaurie Witherwax is the Vice President of Project Management at Worldwide Clinical Trials, which provides drug development services from Early Phase and Bioanalytical Sciences, through Phase II, III and IV. In this episode, Laurie discusses the role of CROs (clinical research organizations).
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Follow us @phaware Engage for a cure: www.phaware.global/donate #phaware #phawareMD291fullphaware global associationVinicio de Jesus Perez, MD - phaware® interview 194Tue, 02 Oct 2018 12:00:00 +0000

In this episode, Dr. Perez discusses genetic markers in a population of Iberian gypsies, who are afflicted with a very severe form of pulmonary hypertension that is called pulmonary veno-occlusive disease. Vinicio de Jesus Perez, MD is Assistant Professor of Medicine (Pulmonary and Critical Care Medicine) at the Stanford Adult PH Clinic where he trains fellows pursuing careers in PH and IPF.

My name is Vinicio de Jesus Perez. I am an assistant professor in the Division of Pulmonary Critical Care Medicine at Stanford University. I'm also a staff physician in the Adult Pulmonary Hypertension Clinic, also at Stanford.

Today I would like to tell you a little bit about one of the ongoing projects that we are engaged in pulmonary hypertension, which has to do with elucidating genetic markers in a population of Iberian gypsies, who are afflicted with a very severe form of pulmonary hypertension called pulmonary veno-occlusive disease (PVOD).

This is a project that we're running in collaboration with colleagues in the Hospital Universitario la Paz in Madrid and the way this happened was that a couple of months ago, I received communication from a graduate student called Jair Tenorio, PhD who become interested after reading one of our publications on use of whole-exome sequencing to discover novel variants in patients with idiopathic pulmonary hypertension that we published around 2014.

He said, "Listen. We have this very interesting patient cohort. We have identified some patients that actually seem to have a familial form of this disease. All of these patients are the product of inter-family marriage. And we believe that there is some new genetic variants. We would like to start a collaboration where we can learn how to use next-generation sequencing strategies and validation tools to discover what the functional impact of these mutation might be."

Jair came to Stanford and stayed in our lab for three months where he had access to our genetic data and learned how to manipulate cell systems to study the effect of candidate mutations. Since returning back to Madrid, he has started do perform whole-exome sequencing of these patients and we have partnered with the U.K. Bridge cohort to collect information about patients with idiopathic and familial PVOD. The questions is: what makes this Iberian gypsy population unique? Are their genetic markers different from those that have been observed in other cohorts in Europe? We have begun developing an international consortium where members of our group will go to Madrid and interact with this Iberian gypsy population. It is worth noting that, historically, this ethnic group has remained aloft due to fear of discrimination and distrust. Luckily, the group in Madrid has established a bond of trust and have gained access to these families. We will share our expertise in next-generation sequencing, bioinformatic strategies, and collaborate to identify what may be responsible for this aggressive phenotype. Ultimately, I think we will obtain a better insight into what drives the development of PVOD from the genetic standpoint.

This is actually a tribe. We're talking about a tribe, we're talking about close to a hundred. And within that, different families. But again, I think it's hard to separate the families because there's been a lot of intermarriage. So, that part, it's a little bit hard. I haven't seen the pedigree analysis that they've done so far. They're going to be quite complicated, compared to what we're used to, when we follow families with heritable pulmonary hypertension.

A lot of these patients have already been seen at the hospitals. We know they actually have severe pulmonary hypertension. Blood samples have been collected, and that's going to be the source of the genetic material that we are going to be using. One interesting tidbit is that we're going to be using techniques to grow out endothelial cells that are circulating from their blood, and also we can potentially use pluripotent stem cell-based strategy to actually make endothelial cells or smooth out amongst the cells to use as a system to model what the pulmonary vasculature may be like. So if these patients have a particular genetic mutation, we could potentially use these cells to see how that affects the endothelium. This is something that has been used and demonstrated to be a feasible strategy by Dr. Marlene Rabinovitch and Dr. Joseph Wu

Historically, most patients with PVOD do really badly because the PH therapies don't really work as well. So what ends up happening is that patients may undergo transplant and at that time, we can capture the tissues and look at architecture, maybe even isolate the cells.

I think that this will also help us identify healthy carriers of mutations and follow them in time using echocardiogram and clinical visits. Think about it: If we know that they are healthy at baseline and they develop PH with time, we may actually get some information about environmental factors or other modifiers that may be of interest in terms of understanding how genetic and environment play a role in developing PVOD.

Jair works with Pilar Escribano, who is the head of the PH group in Hospital Universitario La Paz. She is the clinician who follows these patients. Jair is a post-doc and he works on the genetics side. I think this is going to be really interesting. We are just getting started but I think we have a really big opportunity to study a unique heritable form of pulmonary hypertension.

In this episode, Dr. Perez discusses genetic markers in a population of Iberian gypsies, who are afflicted with a very severe form of pulmonary hypertension that is called pulmonary veno-occlusive disease. Vinicio de Jesus Perez, MD is Assistant Professor of Medicine (Pulmonary and Critical Care Medicine) at the Stanford Adult PH Clinic where he trains fellows pursuing careers in PH and IPF. My name is Vinicio de Jesus Perez. I am an assistant professor in the Division of Pulmonary Critical Care Medicine at Stanford University. I'm also a staff physician in the Adult Pulmonary Hypertension Clinic, also at Stanford.

Today I would like to tell you a little bit about one of the ongoing projects that we are engaged in pulmonary hypertension, which has to do with elucidating genetic markers in a population of Iberian gypsies, who are afflicted with a very severe form of pulmonary hypertension called pulmonary veno-occlusive disease (PVOD).

This is a project that we're running in collaboration with colleagues in the Hospital Universitario la Paz in Madrid and the way this happened was that a couple of months ago, I received communication from a graduate student called Jair Tenorio, PhD who become interested after reading one of our publications on use of whole-exome sequencing to discover novel variants in patients with idiopathic pulmonary hypertension that we published around 2014.

He said, "Listen. We have this very interesting patient cohort. We have identified some patients that actually seem to have a familial form of this disease. All of these patients are the product of inter-family marriage. And we believe that there is some new genetic variants. We would like to start a collaboration where we can learn how to use next-generation sequencing strategies and validation tools to discover what the functional impact of these mutation might be."

Jair came to Stanford and stayed in our lab for three months where he had access to our genetic data and learned how to manipulate cell systems to study the effect of candidate mutations. Since returning back to Madrid, he has started do perform whole-exome sequencing of these patients and we have partnered with the U.K. Bridge cohort to collect information about patients with idiopathic and familial PVOD. The questions is: what makes this Iberian gypsy population unique? Are their genetic markers different from those that have been observed in other cohorts in Europe? We have begun developing an international consortium where members of our group will go to Madrid and interact with this Iberian gypsy population. It is worth noting that, historically, this ethnic group has remained aloft due to fear of discrimination and distrust. Luckily, the group in Madrid has established a bond of trust and have gained access to these families. We will share our expertise in next-generation sequencing, bioinformatic strategies, and collaborate to identify what may be responsible for this aggressive phenotype. Ultimately, I think we will obtain a better insight into what drives the development of PVOD from the genetic standpoint.

This is actually a tribe. We're talking about a tribe, we're talking about close to a hundred. And within that, different families. But again, I think it's hard to separate the families because there's been a lot of intermarriage. So, that part, it's a little bit hard. I haven't seen the pedigree analysis that they've done so far. They're going to be quite complicated, compared to what we're used to, when we follow families with heritable pulmonary hypertension.

A lot of these patients have already been seen at the hospitals. We know they actually have severe pulmonary hypertension. Blood samples have been collected, and that's going to be the source of the genetic material that we are going to be using. One interesting tidbit is that we're going to be using techniques to grow out endothelial cells that are circulating from their blood, and also we can potentially use pluripotent stem cell-based strategy to actually make endothelial cells or smooth out amongst the cells to use as a system to model what the pulmonary vasculature may be like. So if these patients have a particular genetic mutation, we could potentially use these cells to see how that affects the endothelium. This is something that has been used and demonstrated to be a feasible strategy by Dr. Marlene Rabinovitch and Dr. Joseph Wu

Historically, most patients with PVOD do really badly because the PH therapies don't really work as well. So what ends up happening is that patients may undergo transplant and at that time, we can capture the tissues and look at architecture, maybe even isolate the cells.

I think that this will also help us identify healthy carriers of mutations and follow them in time using echocardiogram and clinical visits. Think about it: If we know that they are healthy at baseline and they develop PH with time, we may actually get some information about environmental factors or other modifiers that may be of interest in terms of understanding how genetic and environment play a role in developing PVOD.

Jair works with Pilar Escribano, who is the head of the PH group in Hospital Universitario La Paz. She is the clinician who follows these patients. Jair is a post-doc and he works on the genetics side. I think this is going to be really interesting. We are just getting started but I think we have a really big opportunity to study a unique heritable form of pulmonary hypertension.

]]>fullEpisode 194 - Vinicio A de Jesus Perez, MDVinicio A de Jesus Perez, MD - Pulmonary HypertensionMon, 01 Oct 2018 12:00:00 +0000In this episode, Dr. Perez discusses genetic markers in a population of Iberian gypsies, who are afflicted with a very severe form of pulmonary hypertension that is called pulmonary veno-occlusive disease. Vinicio de Jesus Perez, MD is Assistant Professor of Medicine (Pulmonary and Critical Care Medicine) at the Stanford Adult PH Clinic where he trains fellows pursuing careers in PH and IPF.

]]>In this episode, Dr. Perez discusses genetic markers in a population of Iberian gypsies, who are afflicted with a very severe form of pulmonary hypertension that is called pulmonary veno-occlusive disease. Vinicio de Jesus Perez, MD is Assistant Professor of Medicine (Pulmonary and Critical Care Medicine) at the Stanford Adult PH Clinic where he trains fellows pursuing careers in PH and IPF.

We knew nothing was wrong with Haylee until age three. At her three year old checkup her doctor heard a heart murmur for the first time. He was going to wait a month and then see if he heard it again, and then schedule an echo. In the meantime, her grandfather had been paralyzed and she'd been going to weekly appointments with him. They noticed her blue lips and her blue fingernails, so they urged us to call our pediatrician, which we did.

She had an echo that same week and we found out she has a complete arterial ventricle heart defect as well as pulmonary hypertension. So that very same week, that Friday, she was admitted into St. Children's Hospital and she had a heart catheterization where we learned that she had severe pulmonary hypertension.

We were devastated, angry, like “What is this doctor telling us, that our child's dying? She was just fine a day ago.” So it took a lot of time to sink in, a lot of educating ourselves, reaching out to the PH community before we banded together and said we're going to fight this and do what we can to help save Haylee and everyone else with this.

We started an annual walk that we just had our first walk this year called PH PHun Walk4Hay, which 100% of the proceeds are going to the Robyn Barst Foundation for research for pediatric PH patients. So we just spent a lot of time doing that, getting that whole walk together. And it was actually a very big success this year. We met our goal of 10K and still going. In ends in September.

Research is important for pediatric pulmonary hypertension because there's only one approved treatment for children. Haylee's on that but she's also on two medications that aren't even approved for children, but that's her doctor's only choice to treat her. So we just need more research to find out better ways to treat this and ultimately, of course, a cure.

PH changes everything you ever know. Just know it's definitely going to change everything about your life. You just have to educate yourself, connect with other people, find people that are going to be supportive for you, and then advocate which way is best for you.

I would say her doctor was pretty up front with her about what was going to happen and how serious the disease was. It took a lot of time for us ourselves to let that sink in, but I think he did a good job of letting us know that this is very serious and this is going to change your life and it's going to be complicated.

You just have to take it day by day and then learn it and it will become basically your new way of life, which it has. And Haylee has been our inspiration. So Haylee's the one that keeps us laughing. None of it bothers her and she finds a way to enjoy life no matter what.

We knew nothing was wrong with Haylee until age three. At her three year old checkup her doctor heard a heart murmur for the first time. He was going to wait a month and then see if he heard it again, and then schedule an echo. In the meantime, her grandfather had been paralyzed and she'd been going to weekly appointments with him. They noticed her blue lips and her blue fingernails, so they urged us to call our pediatrician, which we did.

She had an echo that same week and we found out she has a complete arterial ventricle heart defect as well as pulmonary hypertension. So that very same week, that Friday, she was admitted into St. Children's Hospital and she had a heart catheterization where we learned that she had severe pulmonary hypertension.

We were devastated, angry, like “What is this doctor telling us, that our child's dying? She was just fine a day ago.” So it took a lot of time to sink in, a lot of educating ourselves, reaching out to the PH community before we banded together and said we're going to fight this and do what we can to help save Haylee and everyone else with this.

We started an annual walk that we just had our first walk this year called PH PHun Walk4Hay, which 100% of the proceeds are going to the Robyn Barst Foundation for research for pediatric PH patients. So we just spent a lot of time doing that, getting that whole walk together. And it was actually a very big success this year. We met our goal of 10K and still going. In ends in September.

Research is important for pediatric pulmonary hypertension because there's only one approved treatment for children. Haylee's on that but she's also on two medications that aren't even approved for children, but that's her doctor's only choice to treat her. So we just need more research to find out better ways to treat this and ultimately, of course, a cure.

PH changes everything you ever know. Just know it's definitely going to change everything about your life. You just have to educate yourself, connect with other people, find people that are going to be supportive for you, and then advocate which way is best for you.

I would say her doctor was pretty up front with her about what was going to happen and how serious the disease was. It took a lot of time for us ourselves to let that sink in, but I think he did a good job of letting us know that this is very serious and this is going to change your life and it's going to be complicated.

You just have to take it day by day and then learn it and it will become basically your new way of life, which it has. And Haylee has been our inspiration. So Haylee's the one that keeps us laughing. None of it bothers her and she finds a way to enjoy life no matter what.

]]>fullEpisode 193 - Jessica YorkJessica York - Pulmonary HypertensionThu, 27 Sep 2018 12:00:00 +0000Jessica York is a pediatric pulmonary hypertension caregiver to her 6 year old daughter Haylee. In this episode she discusses Hay's diagnosis and the importance of pediatric PH research.

]]>02:52cleanblood,research,heart,pediatric,awareness,therapies,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdiseaseJessica York is a pediatric pulmonary hypertension caregiver to her 6 year old daughter Haylee. In this episode she discusses Hay's diagnosis and the importance of pediatric PH research.
Learn more about clinical trials at: www.phaware.global/clinicaltrials Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware289fullphaware global associationTom Krohn - phaware® interview 192Tue, 25 Sep 2018 12:00:00 +0000

Tom Krohn is the Chief Development Officer at Antidote. In this episode, Tom details a collaboration betweenphaware global association® and Antidote Technologies to accelerate lung disease research. This partnership provides pharmaceutical companies with end-to-end clinical trial recruitment services through an extensive network of patients with chronic lung diseases. Tom is anexperienced executive leader in multiple healthcare and IT settings including sub-Saharan Africa development, US hospital and retail pharmacies, and the pharmaceutical industry. He has a personal passion and commitment to use his talents to serve the marginalized of society.

My name is Tom Krohn. I work at a company called Antidote and we're a digital company helping patients connect to research and researchers connect to patients. Because, unfortunately, there's still a big gap and that's a gap we're trying to close.

My background is in pharmacy. In fact, I grew up in a small-town drugstore in small-town North Dakota. I've been in medical throughout my career, which is about 30 years at this point. It's been a mixture of international development, so I was fortunate to live and work in Madagascar for 10 years, helping build a nonprofit organization there. I came back, got back into hospital practice; I've worked in hospitals and in pharmacies. Fundamentally, I really got convinced that the challenges of health care were really the systems and the management of health care. I felt I had gifts in those areas. I pursued a graduate degree down in Texas and eventually landed in the pharmaceutical industry.

I worked actually at Eli Lilly for 13 years. Throughout that time, it was always in the clinical and regulatory space, especially clinical trials in changing and innovating in how clinical trials were done. Not so much from the scientific point of view, but from an operational point of view, from a business operations point of view and then, importantly in the latter four or five years at Lilly was about external engagement, particularly working with patients differently.

One of the fundamental things we held to was listening, and listening to patients. What's the perspective from a patient? Unfortunately, it's true that once you're in the industry or you're a researcher, you tend to think through the lens of your science, maybe your study, maybe your site or your hospital. It's important to really think through the lens of what it's like on the other side, particularly for patients?

One of the things we clearly heard... we actually brought in patients and did a variety of workshops, etc, was that practical reality is patients are not looking for a specific study. They're not looking for a Lilly study or a Johns Hopkins study. They're looking for a study that might help them. The assumption here is that these are patients who are not having their healthcare met by standard of care. That's not unusual in rare disease, it's not unusual in diseases that are complex. So oncology and cancer is a good example of that, where unfortunately the diagnosis of those diseases is a tough prognosis. It's a tough outcome.

The new therapies are really coming through research. But how do you think of that from a patient point of view? We focused in on really listening and practical aspects are things like, it's hard for patients to find studies that are relevant to them. That's because researchers generally communicate from a scientific point of view only, from a my study point of view and, arguably, a lot of the research is communicated publicly for the sake of compliance; because they have to. That's very important for the transparency initiatives and such. But it's really not to help patients to engage.

So we thought about it and worked on how do we help patients understand their options? We actually built some technology that allowed us to automatically match patients to trials. The patient knows their profile. Why can't we help them find a study, any study? Not only do they want to find them, they want to understand them. How do you engage patients from their point of view? Often it's a nomenclature or a language challenge. Yeah, it's science and there's still a lot of scientific concepts, but there are ways to do things in an interactive way with patients.

An example that I typically use is if I were talking to a nurse, she would ask me if I've had a heart attack before. She would not ask me if I've had a myocardial infarction. So the language we use often is a barrier or it can be used in a way to help patients engage and understand. Ultimately, to us, a big part of that is not only do you have information but what can you do with it? That's how do you take it to the next steps? How do you take it to your doctor to talk about it? How do I understand where the hospitals are that are running these and I can go learn more about these studies? All kinds of next steps. Because it's one thing to be aware, but it's much more to actually engage and be able to follow through.

We focused on that. Ultimately, we've had some good success and Lilly chose to spin out the technology, the platform, the process that we had primarily because Lilly was not in the service business, Lilly is in the drug development business, and it could not represent all the studies out there. If you are serious about serving patients first, you have to give them all the options. So that technology and capabilities was literally sold to a company called TrialReach, which is now Antidote. We just renamed ourselves. Myself and three of my colleagues actually chose to leave Lilly and continue what we started because we believe in what we're doing and there's such a great need here.

So we're involved in bringing that to people. We do that through partners. We're honored to be part of phaware's community and be in a partnership with them to help serve their constituents. But we actually have over 250 of these type of partners. It's because we take on the challenge of bringing order to trials and we make it available to partners like phaware global association®.

It's really to help patients and to close this gap between patients and research. It turns out the problem is just as much on the other side as well. Researchers have a hard time finding patients and we help researchers connect to patients, as well. In fact, if you look at PH, (pulmonary hypertension), there are over 90 studies currently recruiting or about to recruit in the U.S. If you look at how many patients they need, they need 21,000 plus patients just to fill the U.S. studies. Well, there's estimated to be 30,000 PH patients in the U.S. That's over 60%/70%, of all people with a known diagnosis of PH are necessary to participate in the studies.

The point is there's a real demand on the research side and we know that patients with tough diseases like pulmonary hypertension are interested in the new therapies, are interested in new options. We're really here to close the gap between the two.

So Antidote has made a capability we call Match™. We bring order to the clinical trials. We actually start with clinical trials.gov and then we have a team and some capabilities to bring clarity to all those rules. In fact, there are over 2000 ways to say the patient cannot be pregnant. So we have to bring some order to that. So that's what we do and all that's on our side. Fundamentally, a patient, if they came to phaware.global/clinicaltrials and they saw what we call the Match™ widget or the link there, they would be brought into a Match™ environment as if a patient walked into a hospital and wanted to talk to someone about what studies might be relevant and what research. "I'm still struggling and I'm interested in research options."

The interaction with the hospital person, let's say it's a nurse, would not be presenting them with a set of rules. The nurse would start to ask a series of questions. "How old are you? What diagnosis do you have? We've got hospitals around the area, or a network around the area, how far are you willing to travel?" Then they'd start to get into the questions that are more specific to the studies and that's fundamentally what our Match™ does. It presents questions to patients and as they answer questions, depending on their answer, it removes the studies which are no longer relevant to them. So if it's only for pediatrics and you tell us you're 40 years old, those pediatric studies for kids are not relevant to you. As you answer questions, you get fewer and fewer studies, and you can watch the number go down.

What's important is those studies that remain are still relevant to you. So it's not a recommendation engine. What it is, is really especially to take the noise out of the system, is the way I like to think about it and help patients understand their options. Those parameters can be changed: I'm willing to travel farther, all those type of things. But ultimately it's helping patients understand their options. That our Match™ process. Then, if you click through, you can then see which hospitals (we call them sites in the business) are running those [trials]. Because it may not be the hospital here but it may be five miles away or whatever, within the tolerance of your distance. Ultimately, you're getting options to be able to execute and engage in those options.

We are on a journey to cover all studies. We keep our platform up-to-date every night and if there's a change, if there's a new, if there's a subtraction, whatever it is, we're keeping our platform up-to-date because currency really matters and it matters to patients. Our goal is clearly that instead of thinking CT.gov first, you think Antidote because we are basically clinicaltrials.gov but now easy to use, easy to understand and clear calls to action. We don't think about bringing people to Antidote but we think about bringing trials to where the patients are. It's why we have our partnership network. I think over time, you'll see us fill out the rest of the studies across more and more rare diseases in different therapeutic areas, and help patients wherever they are to find the research that's relevant to them.

We've worked closely with phaware global association® leadership, its communities, its researchers, and we're always looking to improve our product, looking to improve our service for patients. That helps both patients, it helps researchers find patients, all that's involved there. So we're glad and honored to be part of the PH community just like we are with other communities. We'd welcome the opportunity to work more closely with you. If you have ideas to improve it, bring it on.

Tom Krohn is the Chief Development Officer at Antidote. In this episode, Tom details a collaboration betweenphaware global association® and Antidote Technologies to accelerate lung disease research. This partnership provides pharmaceutical companies with end-to-end clinical trial recruitment services through an extensive network of patients with chronic lung diseases. Tom is anexperienced executive leader in multiple healthcare and IT settings including sub-Saharan Africa development, US hospital and retail pharmacies, and the pharmaceutical industry. He has a personal passion and commitment to use his talents to serve the marginalized of society. My name is Tom Krohn. I work at a company called Antidote and we're a digital company helping patients connect to research and researchers connect to patients. Because, unfortunately, there's still a big gap and that's a gap we're trying to close.

My background is in pharmacy. In fact, I grew up in a small-town drugstore in small-town North Dakota. I've been in medical throughout my career, which is about 30 years at this point. It's been a mixture of international development, so I was fortunate to live and work in Madagascar for 10 years, helping build a nonprofit organization there. I came back, got back into hospital practice; I've worked in hospitals and in pharmacies. Fundamentally, I really got convinced that the challenges of health care were really the systems and the management of health care. I felt I had gifts in those areas. I pursued a graduate degree down in Texas and eventually landed in the pharmaceutical industry.

I worked actually at Eli Lilly for 13 years. Throughout that time, it was always in the clinical and regulatory space, especially clinical trials in changing and innovating in how clinical trials were done. Not so much from the scientific point of view, but from an operational point of view, from a business operations point of view and then, importantly in the latter four or five years at Lilly was about external engagement, particularly working with patients differently.

One of the fundamental things we held to was listening, and listening to patients. What's the perspective from a patient? Unfortunately, it's true that once you're in the industry or you're a researcher, you tend to think through the lens of your science, maybe your study, maybe your site or your hospital. It's important to really think through the lens of what it's like on the other side, particularly for patients?

One of the things we clearly heard... we actually brought in patients and did a variety of workshops, etc, was that practical reality is patients are not looking for a specific study. They're not looking for a Lilly study or a Johns Hopkins study. They're looking for a study that might help them. The assumption here is that these are patients who are not having their healthcare met by standard of care. That's not unusual in rare disease, it's not unusual in diseases that are complex. So oncology and cancer is a good example of that, where unfortunately the diagnosis of those diseases is a tough prognosis. It's a tough outcome.

The new therapies are really coming through research. But how do you think of that from a patient point of view? We focused in on really listening and practical aspects are things like, it's hard for patients to find studies that are relevant to them. That's because researchers generally communicate from a scientific point of view only, from a my study point of view and, arguably, a lot of the research is communicated publicly for the sake of compliance; because they have to. That's very important for the transparency initiatives and such. But it's really not to help patients to engage.

So we thought about it and worked on how do we help patients understand their options? We actually built some technology that allowed us to automatically match patients to trials. The patient knows their profile. Why can't we help them find a study, any study? Not only do they want to find them, they want to understand them. How do you engage patients from their point of view? Often it's a nomenclature or a language challenge. Yeah, it's science and there's still a lot of scientific concepts, but there are ways to do things in an interactive way with patients.

An example that I typically use is if I were talking to a nurse, she would ask me if I've had a heart attack before. She would not ask me if I've had a myocardial infarction. So the language we use often is a barrier or it can be used in a way to help patients engage and understand. Ultimately, to us, a big part of that is not only do you have information but what can you do with it? That's how do you take it to the next steps? How do you take it to your doctor to talk about it? How do I understand where the hospitals are that are running these and I can go learn more about these studies? All kinds of next steps. Because it's one thing to be aware, but it's much more to actually engage and be able to follow through.

We focused on that. Ultimately, we've had some good success and Lilly chose to spin out the technology, the platform, the process that we had primarily because Lilly was not in the service business, Lilly is in the drug development business, and it could not represent all the studies out there. If you are serious about serving patients first, you have to give them all the options. So that technology and capabilities was literally sold to a company called TrialReach, which is now Antidote. We just renamed ourselves. Myself and three of my colleagues actually chose to leave Lilly and continue what we started because we believe in what we're doing and there's such a great need here.

So we're involved in bringing that to people. We do that through partners. We're honored to be part of phaware's community and be in a partnership with them to help serve their constituents. But we actually have over 250 of these type of partners. It's because we take on the challenge of bringing order to trials and we make it available to partners like phaware global association®.

It's really to help patients and to close this gap between patients and research. It turns out the problem is just as much on the other side as well. Researchers have a hard time finding patients and we help researchers connect to patients, as well. In fact, if you look at PH, (pulmonary hypertension), there are over 90 studies currently recruiting or about to recruit in the U.S. If you look at how many patients they need, they need 21,000 plus patients just to fill the U.S. studies. Well, there's estimated to be 30,000 PH patients in the U.S. That's over 60%/70%, of all people with a known diagnosis of PH are necessary to participate in the studies.

The point is there's a real demand on the research side and we know that patients with tough diseases like pulmonary hypertension are interested in the new therapies, are interested in new options. We're really here to close the gap between the two.

So Antidote has made a capability we call Match™. We bring order to the clinical trials. We actually start with clinical trials.gov and then we have a team and some capabilities to bring clarity to all those rules. In fact, there are over 2000 ways to say the patient cannot be pregnant. So we have to bring some order to that. So that's what we do and all that's on our side. Fundamentally, a patient, if they came to phaware.global/clinicaltrials and they saw what we call the Match™ widget or the link there, they would be brought into a Match™ environment as if a patient walked into a hospital and wanted to talk to someone about what studies might be relevant and what research. "I'm still struggling and I'm interested in research options."

The interaction with the hospital person, let's say it's a nurse, would not be presenting them with a set of rules. The nurse would start to ask a series of questions. "How old are you? What diagnosis do you have? We've got hospitals around the area, or a network around the area, how far are you willing to travel?" Then they'd start to get into the questions that are more specific to the studies and that's fundamentally what our Match™ does. It presents questions to patients and as they answer questions, depending on their answer, it removes the studies which are no longer relevant to them. So if it's only for pediatrics and you tell us you're 40 years old, those pediatric studies for kids are not relevant to you. As you answer questions, you get fewer and fewer studies, and you can watch the number go down.

What's important is those studies that remain are still relevant to you. So it's not a recommendation engine. What it is, is really especially to take the noise out of the system, is the way I like to think about it and help patients understand their options. Those parameters can be changed: I'm willing to travel farther, all those type of things. But ultimately it's helping patients understand their options. That our Match™ process. Then, if you click through, you can then see which hospitals (we call them sites in the business) are running those [trials]. Because it may not be the hospital here but it may be five miles away or whatever, within the tolerance of your distance. Ultimately, you're getting options to be able to execute and engage in those options.

We are on a journey to cover all studies. We keep our platform up-to-date every night and if there's a change, if there's a new, if there's a subtraction, whatever it is, we're keeping our platform up-to-date because currency really matters and it matters to patients. Our goal is clearly that instead of thinking CT.gov first, you think Antidote because we are basically clinicaltrials.gov but now easy to use, easy to understand and clear calls to action. We don't think about bringing people to Antidote but we think about bringing trials to where the patients are. It's why we have our partnership network. I think over time, you'll see us fill out the rest of the studies across more and more rare diseases in different therapeutic areas, and help patients wherever they are to find the research that's relevant to them.

We've worked closely with phaware global association® leadership, its communities, its researchers, and we're always looking to improve our product, looking to improve our service for patients. That helps both patients, it helps researchers find patients, all that's involved there. So we're glad and honored to be part of the PH community just like we are with other communities. We'd welcome the opportunity to work more closely with you. If you have ideas to improve it, bring it on.

]]>fullEpisode 192 - Tom KrohnTom Krohn - Clinical TrialsMon, 24 Sep 2018 12:00:00 +0000Tom Krohn is the Chief Development Officer at Antidote. In this episode, Tom details a collaboration between phaware global association® and Antidote Technologies to accelerate lung disease research. This partnership provides pharmaceutical companies with end-to-end clinical trial recruitment services through an extensive network of patients with chronic lung diseases. Tom is an experienced executive leader in multiple healthcare and IT settings including sub-Saharan Africa development, US hospital and retail pharmacies, and the pharmaceutical industry. He has a personal passion and commitment to use his talents to serve the marginalized of society.

]]>Tom Krohn is the Chief Development Officer at Antidote. In this episode, Tom details a collaboration between phaware global association® and Antidote Technologies to accelerate lung disease research. This partnership provides pharmaceutical companies with end-to-end clinical trial recruitment services through an extensive network of patients with chronic lung diseases. Tom is an experienced executive leader in multiple healthcare and IT settings including sub-Saharan Africa development, US hospital and retail pharmacies, and the pharmaceutical industry. He has a personal passion and commitment to use his talents to serve the marginalized of society.

At the age of three, Maleen Fischer was diagnosed with pulmonary hypertension and, as there was no treatment for this fatal disease at the time in Austria, doctors sent her home. Her life expectancy was only a few years. Now, 20 years later, Maleen discusses early diagnosis, the joy of graduating college and the importance of global awareness.

My name is Maleen Fischer. I'm from Vienna, Austria. When I was very little, my mom, early on, she noticed when she was breastfeeding me - she just described it as thinking that I was out of breath, and she kind of from the get go, after I was born, she knew something was up. We hurried from doctor to doctor until I was diagnosed (with idiopathic pulmonary hypertension) when I was around three years old.

Back in Vienna, at that time, nobody really knew what to do with somebody with that diagnosis, and so they would just kind of say, “Well, you would have to see how it goes,” but they did not give me a very long life expectancy. My father, as determined as he is, and was specifically at that time, he found a very good hospital in New York that was working on treating pulmonary hypertension, and we went there, and we met with a wonderful doctor (Robyn J. Barst, MD) that has since passed away, unfortunately. She put me on an IV treatment that I'm still on to this day.

Now I'm 23 years old, my birthday was in May. I'm doing very well, so I can be very thankful for my family who really didn't want to give up. Now, when I still think back to New York, it's a very hate/love kind of relationship to just think back to that time. On one hand, it's a beautiful city, and I was always so impressed with everything. My parents, obviously, also tried to make it a great time, always. And, on the other hand, having the doctor's visits, being put on a pump, on an IV system, and I just remember when we were picking out the first bags and backpacks to put the pump in. This kind of journey, to kind of adapt to a whole new lifestyle.

I find myself, in this situation, very lucky that I was diagnosed so early on, so I could get used to this lifestyle for many years. I would assume it's very difficult to be diagnosed at a much later age. I must say I'm very lucky, I've always had friends that were really respecting and accepting of the whole situation. My neighbor, we kind of grew up together, and she knew me even before the pump, as little babies.

I did not go to Kindergarten. I was homeschooled throughout those years and then all the way to high school. And then, what I did in high school was I went, for the first time, into a real school, because with pulmonary hypertension we take into account how many steps we walk during school, and also how young the children are and how understanding they are of not ripping away a backpack. When high school time came around we said, “Okay, this would be the time where we could try out to actually go to a school." I went to a very small school in Florida, and it was completely flat, so no stairs and a very small amount of students. This was the first time I was actually in high school and experiencing other teenagers. It was kind of funny because it really incorporated all the high school drama, first boyfriend love story, whatever, in a very short amount of time. I actually felt very normal during that time, I have to say.

When I was diagnosed, this disease was not known, and so my dad and I, we really did a lot of awareness work; and it started just right after I was put on that pump. We did all types of newspaper reports on it, or documentations. Always my goal was to really get the word out there about this disease, because during our time of working in awareness, we've had so many stories of people coming to us saying, “Because of you, because I saw this show, or because I saw this report, or this news article, I went to the doctor and I actually have pulmonary hypertension”.

When I went into my teenage years I thought, “What can I really do to kind of emphasize that even more? What could give people hope at the same time as kind of give other people a perspective of what pulmonary hypertension really means, and what it does to a life?” From the good parts to the bad parts. That, in the end, was essentially how I ended up writing a book. It really also shows my journey as a person, because the book changed drastically when a few certain situations happened within those, let's say two and a half to three years I was working on it. When it started out, I was speaking about how it was like to go to New York, being put on this medication, living with this medication, going through high school with this medication.

I believe it was in my last year of high school, I had an extremely bad case of an appendicitis and it was overlooked. I can't really figure out, or can't remember the medical term for it, but I had an abnormal appendix so you could not see it on an X-ray and I was sent home with antibiotics, and a day later my appendix ruptured. I was intubated, which we all know is really bad for pulmonary hypertension patients, anybody but specifically us. I was just doing really bad. I barely remember that time, to be honest. I had a sepsis, of course, multiple blood transfusions. This was a time where, of course, throughout my lifetime there was always a lot of uncertainty; but at this point you could really feel, “Okay, I am definitely not invincible, and things happen and things can get really bad.”

I somehow recovered from that, and then, what you have afterwards, the effects, trying to bring your saturation up again, trying to be able to breathe comfortably and with enough saturation, without oxygen masks and the compression masks. And this was really the turning point where I figured, “Okay, this is where I add on to the book, and this is also my way of coping.”

After I finished high school in Florida, and I also did my tests and certifications in Austria, then I looked at colleges. I looked at options that I had, and I wanted to stay in Vienna just because I had a good net of doctors here, and I felt very comfortable here also with my friends and family, and so I found a great college here. This was also the first time that I was a little more detached from my parents. It felt very nice. I know a lot of people say that college is the best time of your life. I don't know about that exactly, but it certainly was a wonderful time and I met so many great people.

It had the same effect to me as high school did, a little bit. It made me feel very normal. In between that, I had some issues with my condition and with the pump, all sorts of things, random things you can think about; but then I also had normal problems like studying for tests, or just mundane things that you really appreciate more, I feel like.

In May, I had my graduation. I was very happy I could graduate with a lot of my friends, actually. My best friend that I had known also still before the pump, and she has a very similar name as mine, it's Marlene, and mine is Maleen, so people would always just call us “M&Ms” instead of having to say the whole name. It was very nice because we would joke around as kids that we would graduate from the same college, and we did. We ended up doing that, and it never even seemed like that was an option.

I worked very hard for those four years, I was very ambitious, and I figured, “If you go on this journey, you're going to college, you might as well just try your best.” Generally, I have this type of personality that I just need to at least try as much as I can in order to feel like, “Okay, if I give it my all if it doesn't work out then I guess it wasn't meant to be.” Luckily, it was good. I graduated with high honors and we had just a great time graduating all together and it was a really, really nice experience. I smile back just thinking about it, and I'm really also just going to miss the whole experience, but I think grad school is definitely going to be a new chapter. Also, at one point, I want to hopefully be working more. I'm looking forward to that.

The advice I would have for somebody that would be newly diagnosed, walking out of a doctor's office, I would say sometimes not everything that doctors tell you has to be taken exactly word for word. Prove them wrong, and so if you get a bad diagnosis you don't have to take it as it is. Don't put your life to a complete stop. That doesn't mean that you shouldn't take the time a little bit to figure out what to do next, but keep going with the things that you want to do. Not everything might still be an option for you, but there are things that you can still do.

Everybody has so many choices in life. That's a beautiful thing about life. You have so many choices, and if one thing doesn't work out, it doesn't mean that that is the only thing you need to limit yourself to. You can find an alternative. Just go for it, and do it, because even if you feel bad sometimes, that success, or sometimes just doing something that makes you happy, it will make you feel better.

It's always so important to create awareness, and awareness does not need to be going on national television or doing crazy things. You can just simply, honestly tell people that you meet about this disease. Bring it to the attention of other people, and that can already make a difference. That will also make it easier for them to understand, because people are just naturally curious.

And, the second thing would be, if you find yourself in a hole or you think that something's wrong with you because you're just super depressed and other people that you see are coping with the disease so well... It's like Instagram, honestly, most of the time you see really positive things about other people, and even people that have been diagnosed will maybe just tell you the positive things, or tell you that they're coping well. If people feel like they're not doing well just because sometimes they're depressed or sometimes they don't really know what to do anymore because of the disease, I think everybody feels like that every now and then. Just never feel like an outsider because of that.

At the age of three, Maleen Fischer was diagnosed with pulmonary hypertension and, as there was no treatment for this fatal disease at the time in Austria, doctors sent her home. Her life expectancy was only a few years. Now, 20 years later, Maleen discusses early diagnosis, the joy of graduating college and the importance of global awareness.

My name is Maleen Fischer. I'm from Vienna, Austria. When I was very little, my mom, early on, she noticed when she was breastfeeding me - she just described it as thinking that I was out of breath, and she kind of from the get go, after I was born, she knew something was up. We hurried from doctor to doctor until I was diagnosed (with idiopathic pulmonary hypertension) when I was around three years old.

Back in Vienna, at that time, nobody really knew what to do with somebody with that diagnosis, and so they would just kind of say, “Well, you would have to see how it goes,” but they did not give me a very long life expectancy. My father, as determined as he is, and was specifically at that time, he found a very good hospital in New York that was working on treating pulmonary hypertension, and we went there, and we met with a wonderful doctor (Robyn J. Barst, MD) that has since passed away, unfortunately. She put me on an IV treatment that I'm still on to this day.

Now I'm 23 years old, my birthday was in May. I'm doing very well, so I can be very thankful for my family who really didn't want to give up. Now, when I still think back to New York, it's a very hate/love kind of relationship to just think back to that time. On one hand, it's a beautiful city, and I was always so impressed with everything. My parents, obviously, also tried to make it a great time, always. And, on the other hand, having the doctor's visits, being put on a pump, on an IV system, and I just remember when we were picking out the first bags and backpacks to put the pump in. This kind of journey, to kind of adapt to a whole new lifestyle.

I find myself, in this situation, very lucky that I was diagnosed so early on, so I could get used to this lifestyle for many years. I would assume it's very difficult to be diagnosed at a much later age. I must say I'm very lucky, I've always had friends that were really respecting and accepting of the whole situation. My neighbor, we kind of grew up together, and she knew me even before the pump, as little babies.

I did not go to Kindergarten. I was homeschooled throughout those years and then all the way to high school. And then, what I did in high school was I went, for the first time, into a real school, because with pulmonary hypertension we take into account how many steps we walk during school, and also how young the children are and how understanding they are of not ripping away a backpack. When high school time came around we said, “Okay, this would be the time where we could try out to actually go to a school." I went to a very small school in Florida, and it was completely flat, so no stairs and a very small amount of students. This was the first time I was actually in high school and experiencing other teenagers. It was kind of funny because it really incorporated all the high school drama, first boyfriend love story, whatever, in a very short amount of time. I actually felt very normal during that time, I have to say.

When I was diagnosed, this disease was not known, and so my dad and I, we really did a lot of awareness work; and it started just right after I was put on that pump. We did all types of newspaper reports on it, or documentations. Always my goal was to really get the word out there about this disease, because during our time of working in awareness, we've had so many stories of people coming to us saying, “Because of you, because I saw this show, or because I saw this report, or this news article, I went to the doctor and I actually have pulmonary hypertension”.

When I went into my teenage years I thought, “What can I really do to kind of emphasize that even more? What could give people hope at the same time as kind of give other people a perspective of what pulmonary hypertension really means, and what it does to a life?” From the good parts to the bad parts. That, in the end, was essentially how I ended up writing a book. It really also shows my journey as a person, because the book changed drastically when a few certain situations happened within those, let's say two and a half to three years I was working on it. When it started out, I was speaking about how it was like to go to New York, being put on this medication, living with this medication, going through high school with this medication.

I believe it was in my last year of high school, I had an extremely bad case of an appendicitis and it was overlooked. I can't really figure out, or can't remember the medical term for it, but I had an abnormal appendix so you could not see it on an X-ray and I was sent home with antibiotics, and a day later my appendix ruptured. I was intubated, which we all know is really bad for pulmonary hypertension patients, anybody but specifically us. I was just doing really bad. I barely remember that time, to be honest. I had a sepsis, of course, multiple blood transfusions. This was a time where, of course, throughout my lifetime there was always a lot of uncertainty; but at this point you could really feel, “Okay, I am definitely not invincible, and things happen and things can get really bad.”

I somehow recovered from that, and then, what you have afterwards, the effects, trying to bring your saturation up again, trying to be able to breathe comfortably and with enough saturation, without oxygen masks and the compression masks. And this was really the turning point where I figured, “Okay, this is where I add on to the book, and this is also my way of coping.”

After I finished high school in Florida, and I also did my tests and certifications in Austria, then I looked at colleges. I looked at options that I had, and I wanted to stay in Vienna just because I had a good net of doctors here, and I felt very comfortable here also with my friends and family, and so I found a great college here. This was also the first time that I was a little more detached from my parents. It felt very nice. I know a lot of people say that college is the best time of your life. I don't know about that exactly, but it certainly was a wonderful time and I met so many great people.

It had the same effect to me as high school did, a little bit. It made me feel very normal. In between that, I had some issues with my condition and with the pump, all sorts of things, random things you can think about; but then I also had normal problems like studying for tests, or just mundane things that you really appreciate more, I feel like.

In May, I had my graduation. I was very happy I could graduate with a lot of my friends, actually. My best friend that I had known also still before the pump, and she has a very similar name as mine, it's Marlene, and mine is Maleen, so people would always just call us “M&Ms” instead of having to say the whole name. It was very nice because we would joke around as kids that we would graduate from the same college, and we did. We ended up doing that, and it never even seemed like that was an option.

I worked very hard for those four years, I was very ambitious, and I figured, “If you go on this journey, you're going to college, you might as well just try your best.” Generally, I have this type of personality that I just need to at least try as much as I can in order to feel like, “Okay, if I give it my all if it doesn't work out then I guess it wasn't meant to be.” Luckily, it was good. I graduated with high honors and we had just a great time graduating all together and it was a really, really nice experience. I smile back just thinking about it, and I'm really also just going to miss the whole experience, but I think grad school is definitely going to be a new chapter. Also, at one point, I want to hopefully be working more. I'm looking forward to that.

The advice I would have for somebody that would be newly diagnosed, walking out of a doctor's office, I would say sometimes not everything that doctors tell you has to be taken exactly word for word. Prove them wrong, and so if you get a bad diagnosis you don't have to take it as it is. Don't put your life to a complete stop. That doesn't mean that you shouldn't take the time a little bit to figure out what to do next, but keep going with the things that you want to do. Not everything might still be an option for you, but there are things that you can still do.

Everybody has so many choices in life. That's a beautiful thing about life. You have so many choices, and if one thing doesn't work out, it doesn't mean that that is the only thing you need to limit yourself to. You can find an alternative. Just go for it, and do it, because even if you feel bad sometimes, that success, or sometimes just doing something that makes you happy, it will make you feel better.

It's always so important to create awareness, and awareness does not need to be going on national television or doing crazy things. You can just simply, honestly tell people that you meet about this disease. Bring it to the attention of other people, and that can already make a difference. That will also make it easier for them to understand, because people are just naturally curious.

And, the second thing would be, if you find yourself in a hole or you think that something's wrong with you because you're just super depressed and other people that you see are coping with the disease so well... It's like Instagram, honestly, most of the time you see really positive things about other people, and even people that have been diagnosed will maybe just tell you the positive things, or tell you that they're coping well. If people feel like they're not doing well just because sometimes they're depressed or sometimes they don't really know what to do anymore because of the disease, I think everybody feels like that every now and then. Just never feel like an outsider because of that.

]]>fullEpisode 191 - Maleen FischerMaleen Fischer - Pulmonary HypertensionThu, 20 Sep 2018 12:00:00 +0000At the age of three, Maleen Fischer was diagnosed with pulmonary hypertension and, as there was no treatment for this fatal disease at the time in Austria, doctors sent her home. Her life expectancy was only a few years. Now, 20 years later, Maleen discusses early diagnosis, the joy of graduating college and the importance of global awareness.

Click here to learn more about Maleen's book: As If There Were No Tomorrow: Hope Springs Eternal.

]]>At the age of three, Maleen Fischer was diagnosed with pulmonary hypertension and, as there was no treatment for this fatal disease at the time in Austria, doctors sent her home. Her life expectancy was only a few years. Now, 20 years later, Maleen discusses early diagnosis, the joy of graduating college and the importance of global awareness.

Click here to learn more about Maleen's book: As If There Were No Tomorrow: Hope Springs Eternal.

]]>10:00cleanblood,research,heart,awareness,therapies,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdiseaseAt the age of three, Maleen Fischer was diagnosed with pulmonary hypertension and, as there was no treatment for this fatal disease at the time in Austria, doctors sent her home. Her life expectancy was only a few years. Now, 20 years later, Maleen discusses early diagnosis, the joy of graduating college and the importance of global awareness.
Learn more about pulmonary hypertension at www.phaware365.global. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin #phaware 287fullphaware global association Victor F. Tapson, MD - phaware® interview 190Tue, 18 Sep 2018 12:00:00 +0000

Victor F. Tapson, MD discusses the PERT Consortium, a network of multidisciplinary pulmonary embolism response teams working to improve the care of pts w severe PE. The PERT Consortium intends to guide and influence pulmonary embolism (PE) care and research in institutions across the U.S. and will be the driving force behind increased survival rates and the future of PE treatment.

Today I want to talk about the concept of the pulmonary embolism response team [PERT] and how it interfaces with CTEPH.

We think of this as a concept of a patient coming in the hospital with acute pulmonary embolism and a team of people trying to decide how to best handle things. And that's really what a PERT is. We also follow patients long term. When we see someone in the hospital with acute pulmonary embolism, they have our contact information, we see them back in the clinic, usually within two weeks. We see them again at three months, and we follow them forever. And I think that's very important. You get your CTEPH population referrals often as full blown CTEPH patients with bad pulmonary hypertension. They're seen by a pulmonologist or cardiologist, who figure out they've got PH and they may figure out they have CTEPH. Either way, they're sent to you and you diagnose them with CTEPH and try to get them treated.

The other pathway that's very important is of course to realize everyone with CTEPH had pulmonary embolism at one point. So when those patients come to our clinic, we're following them not only to see how they're doing acutely, but to keep in mind there are certain risk factors for CTEPH. Splenectomy, perhaps the size of the PE. Again, certain things that may put patients at higher risk for CTEPH. But either way, we follow them along, we follow their symptoms. In certain cases we repeat imaging. We always make sure the echocardiogram goes back to normal. But this is a great way with a big pulmonary embolism clinic like we have at Cedars, if you're following hundreds and hundreds of acute PE patients, you're going to get CTEPH eventually and you're not going to miss them if they're in a clinic like that.

So I'd certainly encourage patients with acute pulmonary embolism to be seen in a PE clinic, if you have one like we have at Cedars. If you don't, the family practitioner, internist, whoever might follow that patient, probably ought to refer them to an expert once a year. Have them talk to them about their breathing, consider duration of anticoagulation. Understand that we have new data now with Amplify Extend and Einstein Choice for longer therapy of anticoagulation with lower doses of either 10 milligrams of rivaroxaban or 2.5 twice daily of apixaban. Bottom line is we've got a lot of new things going on and people need to know about that, so consider after your PERT patients are seen in the hospital, make sure they get long term follow up, you may pick up some CTEPH that way.

I'm President right now of the PE Response Team Consortium. This is a 501(c)(3), organized initially at Mass General Hospital with the help of individuals like Ken Rosenfield, and the concept's really evolved and caught on. Having a PE response team in your hospital and also getting that PE response team to join our national consortium, which actually is international. We've had folks from Brazil and Shaoping Lee from China, some great organizations from South America. So we're becoming international. And this PERT concept involves several things. One is making sure you have a fully operational PERT at your hospital, but also getting involved with and getting the word out about acute pulmonary embolism, PE research, clinical trials. With the PERT Consortium, we're organizing clinical trials, endorsing certain trials, coming up with ideas. We have a number of just fantastic committees. We have an amazing education committee, protocol committee, governance committee, communications committee, research committee.

We have a great database that's finally getting off the ground. We've moved our database from Mass General to the Boston Clinical Research Institute with Mike Gibson. We're very excited about that. This REDCap database is going to help us get quality assurance data from around the country as well as a lot of research data. So we're going to make some big moves in the next year or so with all the data we're gathering, and try to determine, based on a lot of PERT data, what the best studies are to do next, where we stand and what the major problems in pulmonary embolism are.

This disease continues to kill 100,000 people per year in the United States. Probably affects 800,000 to 900,000 per year in the United States. It's a bad disease that not only leads to death in some cases, but can lead to CTEPH and other complications. So the PERT Consortium is really trying to work hard to get a multidisciplinary group of individuals from cardiology, pulmonary, radiology, interventional radiology, ED, vascular surgery, vascular medicine, multidisciplinary groups, CT surgery, so that we can work together to come up with the best solutions for pulmonary embolism.

Victor F. Tapson, MD discusses the PERT Consortium, a network of multidisciplinary pulmonary embolism response teams working to improve the care of pts w severe PE. The PERT Consortium intends to guide and influence pulmonary embolism (PE) care and research in institutions across the U.S. and will be the driving force behind increased survival rates and the future of PE treatment.

Today I want to talk about the concept of the pulmonary embolism response team [PERT] and how it interfaces with CTEPH.

We think of this as a concept of a patient coming in the hospital with acute pulmonary embolism and a team of people trying to decide how to best handle things. And that's really what a PERT is. We also follow patients long term. When we see someone in the hospital with acute pulmonary embolism, they have our contact information, we see them back in the clinic, usually within two weeks. We see them again at three months, and we follow them forever. And I think that's very important. You get your CTEPH population referrals often as full blown CTEPH patients with bad pulmonary hypertension. They're seen by a pulmonologist or cardiologist, who figure out they've got PH and they may figure out they have CTEPH. Either way, they're sent to you and you diagnose them with CTEPH and try to get them treated.

The other pathway that's very important is of course to realize everyone with CTEPH had pulmonary embolism at one point. So when those patients come to our clinic, we're following them not only to see how they're doing acutely, but to keep in mind there are certain risk factors for CTEPH. Splenectomy, perhaps the size of the PE. Again, certain things that may put patients at higher risk for CTEPH. But either way, we follow them along, we follow their symptoms. In certain cases we repeat imaging. We always make sure the echocardiogram goes back to normal. But this is a great way with a big pulmonary embolism clinic like we have at Cedars, if you're following hundreds and hundreds of acute PE patients, you're going to get CTEPH eventually and you're not going to miss them if they're in a clinic like that.

So I'd certainly encourage patients with acute pulmonary embolism to be seen in a PE clinic, if you have one like we have at Cedars. If you don't, the family practitioner, internist, whoever might follow that patient, probably ought to refer them to an expert once a year. Have them talk to them about their breathing, consider duration of anticoagulation. Understand that we have new data now with Amplify Extend and Einstein Choice for longer therapy of anticoagulation with lower doses of either 10 milligrams of rivaroxaban or 2.5 twice daily of apixaban. Bottom line is we've got a lot of new things going on and people need to know about that, so consider after your PERT patients are seen in the hospital, make sure they get long term follow up, you may pick up some CTEPH that way.

I'm President right now of the PE Response Team Consortium. This is a 501(c)(3), organized initially at Mass General Hospital with the help of individuals like Ken Rosenfield, and the concept's really evolved and caught on. Having a PE response team in your hospital and also getting that PE response team to join our national consortium, which actually is international. We've had folks from Brazil and Shaoping Lee from China, some great organizations from South America. So we're becoming international. And this PERT concept involves several things. One is making sure you have a fully operational PERT at your hospital, but also getting involved with and getting the word out about acute pulmonary embolism, PE research, clinical trials. With the PERT Consortium, we're organizing clinical trials, endorsing certain trials, coming up with ideas. We have a number of just fantastic committees. We have an amazing education committee, protocol committee, governance committee, communications committee, research committee.

We have a great database that's finally getting off the ground. We've moved our database from Mass General to the Boston Clinical Research Institute with Mike Gibson. We're very excited about that. This REDCap database is going to help us get quality assurance data from around the country as well as a lot of research data. So we're going to make some big moves in the next year or so with all the data we're gathering, and try to determine, based on a lot of PERT data, what the best studies are to do next, where we stand and what the major problems in pulmonary embolism are.

This disease continues to kill 100,000 people per year in the United States. Probably affects 800,000 to 900,000 per year in the United States. It's a bad disease that not only leads to death in some cases, but can lead to CTEPH and other complications. So the PERT Consortium is really trying to work hard to get a multidisciplinary group of individuals from cardiology, pulmonary, radiology, interventional radiology, ED, vascular surgery, vascular medicine, multidisciplinary groups, CT surgery, so that we can work together to come up with the best solutions for pulmonary embolism.

]]>Victor F. Tapson, MD discusses the PERT Consortium, a network of multidisciplinary pulmonary embolism response teams working to improve the care of pts w severe PE. The PERT Consortium intends to guide and influence pulmonary embolism (PE) care and research in institutions across the U.S. and will be the driving force behind increased survival rates and the future of PE treatment.

Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center explains the basics of clinical trials in part 2 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Dr. McLaughlin is an editorial board member of Chest, a Guest Editor for the Journal of the American College of Cardiology, and past Editor-in-Chief of Advances in Pulmonary Hypertension.

I'm Vallerie McLaughlin. I'm a cardiologist at the University of Michigan and my area of expertise is pulmonary hypertension.

Today, we'll go a little deeper into clinical trials.

An observational trial is just that, it observes what happens to a patient in a certain condition. Registries, for example, are observational trials. And often times there are Phase IV registries that occur after medications have been approved. If you think about it sometimes a limited number of patients are enrolled in the trials that lead to the FDA approval of the agent but other side effects may pop up once patients are exposed. So registries are a great way to look for those less common side effects. So an observational registry would be a situation in which a patient is prescribed a therapy by their physician that is commercially indicated, and the patient consents to allow their data while on therapy to be obtained for the purpose of the registry. Maybe they'll pick up a side effect that was not anticipated or learn something else when a large number of patients are exposed to that agent. So, that's an observational trial.

An interventional clinical trial is a situation where we're trying to learn about a medication and measure something specific. So the patient is often randomized and there are measurements of some efficacy end point. There are study specific procedures that are done to answer a question. In an observational study there's often no study specific procedure. We just record what's happening that's clinically indicated. The interventional trial has a specific question.

Many clinical trials are placebo controlled. Often the Phase III pivotal trials that are used to get FDA approval of an agent are placebo controlled. But there may be instances where earlier phased trials don't have placebo control. They may just test different doses of the same agent. Sometimes the Phase IV trials aren't placebo controlled. Randomization is an important part of clinical trials. What randomization means is that once a study subject consents they are randomized or randomly assigned to a group to either receive placebo or a medication or to receive this dose of medications or that dose of medication. And it's a way that we can statically assess what the intervention is doing.

So the patient has a very important role in clinical trials. We want patients to participate in clinical trials who are invested in the trial and agree with what is going to happen. That's part of what we call the consent process. They need to understand what is going to happen in the trial. What the extra visits are. What the risks of the medication are. It's important for the patient to read that consent thoroughly and agree to participate with what is going to happen.

Many clinical trials are sponsored by companies or government agencies that want a specific question answered. For example, many trials are investigational drugs. So they're often sponsored by pharmaceutical companies that have been developing this drug for a certain indication. So their role is to often work with a scientific body to put together a protocol, a statistical analysis plan. Basically the plan for the trial. And then provide the resources to do the trial.

A clinical research organization is sometimes involved in clinical trials to help smooth the process. A clinical research organization often serves as the intermediary between the sponsor and the study site. And provides the study site with protocols with the case report forms. They often monitor the site to make sure they're doing the trial properly. And they help resolve any queries, any questions about data that has been obtained.

So the principle investigator is very important. Is really ultimately responsible for the conduct of the clinical trial at that site. The principle investigator at an institution really takes the responsibility for not only what they do as the investigator but what everyone else does. What the study coordinators do. What the investigational drug services do. They really are the quarterback for the clinical trial at that site. And it's their responsibility to make sure that the trial is done ethically and in accordance with good clinical practices that are accepted worldwide.

The institutional review board is very important to ensure patient safety. Many institutions have their own institutional review board. There are some contract institutional review boards that different investigators can work with. The institutional review board reviews the study, looks at it primarily with patient safety in mind. They want to make sure that this is a reasonable, safe, ethical study for the patient to participate in. And they want to make sure the patient understands the risk. The institutional review board looks over the consent and really makes sure it is explaining to patient what exactly will happen in that study. So every investigator needs and IRB, institutional review board approval to start a study at their site. And then other things need to be reported to the IRB as well, if there's a problem, if there's an adverse event that's unexpected, that needs to go to the IRB so they can determine if it's safe to continue exposing patients to the study.

Clinical trials are very regimented and have very strict protocols for what happens. One of the most important criteria or things in the protocol are the entry and exclusion criteria for that trial. Clinical trials often want to answer a specific question in a specific patient population. And the inclusion criteria help define that patient population. It may be patients with a disease that have certain criteria based on how advanced they are, how many years they've had the disease, how sick they are, a number of different criteria like that. And then there's often exclusion criteria as well. We don't want patients who have too many other comorbidities or something that might limit their life or exposure to drug or obviously an important one, is they're competent to participate in the trial. So it may have exclusion criteria about cooperation, mental health issues, drug abuse, that sort of thing. So it's important to make sure that all the patients studied in the trial are similar to answer the question in this defined population.

The participation in a clinical trial is something that is decided upon by both the patient and the investigator. So remember, the patients need to willing participate. This is a choice that the patients have. They have to decide if they want to go into a clinical trial. And even once the patient decides that they're willing to go into the clinical trial, the investigator needs to make sure that they meet all the inclusion and exclusion criteria. So they both need to be on the same page.

It's very important for a patient to know all the options they have for their care before participating in a clinical trial. It needs to be a totally voluntary decision. It shouldn't change the care that you receive by your physician. So one thing that patients need to know is that this is ultimately their decision to participate. And then, of course, it's important to know the details about the trial. The responsibilities, the risks of the trial related medication or any trial related procedures.

There are many reasons a clinical trial can be successful or unsuccessful. It can be unsuccessful if the medication truly doesn't work and that's one of the reasons we do clinical trials. We want to understand if a medication works in a particular patient population. So that's an important reason that it might not be successful. Other reasons might be that it was designed poorly. A poor end point was chosen. There could be a number of different other issues.

Sometimes we don't know the results of clinical trials for a long time. A patient might be enrolled at a site early in the trial and it may take another year or two or three to finish enrolling the clinical trial. So sometimes it seems like they don't know the results but it may just be because the trial's not over. Often times the investigator can share the results of the trial with the patients once all is said and done.

There are many clinical trials for patients with diseases and there are other options for patients who don't have specific disease. Many clinical trials want to look at healthy controls to see the effect of a therapy in those patients as well. And then of course the Phase I trials often involve normal controls. Usually when a patient participates in a clinical trial it should not cost them any money and in fact all of the procedures that are specifically for the trial should be covered as part of the clinical trial.

Clinical trials are ways to learn about therapies. And sometimes we don't know the effects of the therapy so there are potentially situations where there might be side effects of a therapy or side effects of a procedure that the patient might consider is not the safest option for them and that's an important thing for the patient to consider before they go into a clinical trial. Remember by the time a patient is approached about a clinical trial, usually the FDA has signed off on for their study and of course the IRB has reviewed it and has assessed the risks of the study. Sometimes we take risks in order to get benefits as well. And this has to be balanced with patients, their diagnosis and how sick they are.

Clinical trials are so important because it's our way to learn about therapies that we want to get out to patients. For example, in the disease that I take care of, it's a rare disease, the very first therapy was approved the year I started practicing, 1995, that was the first time we had anything for patients. Now, I tell them about a dozen different options that they have to treat their disease. And they're doing so much better than they were in 1995. And we wouldn't have those therapies if it weren't for all of the patients who participated in clinical trials before that.

While clinical trials are voluntary for any patient it is important for a patient to consider doing good for their community and their disease state and how their participation in the clinical trial might help further knowledge and lead to therapies for patients like them down the road.

Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center explains the basics of clinical trials in part 2 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Dr. McLaughlin is an editorial board member of Chest, a Guest Editor for the Journal of the American College of Cardiology, and past Editor-in-Chief of Advances in Pulmonary Hypertension.

I'm Vallerie McLaughlin. I'm a cardiologist at the University of Michigan and my area of expertise is pulmonary hypertension.

Today, we'll go a little deeper into clinical trials.

An observational trial is just that, it observes what happens to a patient in a certain condition. Registries, for example, are observational trials. And often times there are Phase IV registries that occur after medications have been approved. If you think about it sometimes a limited number of patients are enrolled in the trials that lead to the FDA approval of the agent but other side effects may pop up once patients are exposed. So registries are a great way to look for those less common side effects. So an observational registry would be a situation in which a patient is prescribed a therapy by their physician that is commercially indicated, and the patient consents to allow their data while on therapy to be obtained for the purpose of the registry. Maybe they'll pick up a side effect that was not anticipated or learn something else when a large number of patients are exposed to that agent. So, that's an observational trial.

An interventional clinical trial is a situation where we're trying to learn about a medication and measure something specific. So the patient is often randomized and there are measurements of some efficacy end point. There are study specific procedures that are done to answer a question. In an observational study there's often no study specific procedure. We just record what's happening that's clinically indicated. The interventional trial has a specific question.

Many clinical trials are placebo controlled. Often the Phase III pivotal trials that are used to get FDA approval of an agent are placebo controlled. But there may be instances where earlier phased trials don't have placebo control. They may just test different doses of the same agent. Sometimes the Phase IV trials aren't placebo controlled. Randomization is an important part of clinical trials. What randomization means is that once a study subject consents they are randomized or randomly assigned to a group to either receive placebo or a medication or to receive this dose of medications or that dose of medication. And it's a way that we can statically assess what the intervention is doing.

So the patient has a very important role in clinical trials. We want patients to participate in clinical trials who are invested in the trial and agree with what is going to happen. That's part of what we call the consent process. They need to understand what is going to happen in the trial. What the extra visits are. What the risks of the medication are. It's important for the patient to read that consent thoroughly and agree to participate with what is going to happen.

Many clinical trials are sponsored by companies or government agencies that want a specific question answered. For example, many trials are investigational drugs. So they're often sponsored by pharmaceutical companies that have been developing this drug for a certain indication. So their role is to often work with a scientific body to put together a protocol, a statistical analysis plan. Basically the plan for the trial. And then provide the resources to do the trial.

A clinical research organization is sometimes involved in clinical trials to help smooth the process. A clinical research organization often serves as the intermediary between the sponsor and the study site. And provides the study site with protocols with the case report forms. They often monitor the site to make sure they're doing the trial properly. And they help resolve any queries, any questions about data that has been obtained.

So the principle investigator is very important. Is really ultimately responsible for the conduct of the clinical trial at that site. The principle investigator at an institution really takes the responsibility for not only what they do as the investigator but what everyone else does. What the study coordinators do. What the investigational drug services do. They really are the quarterback for the clinical trial at that site. And it's their responsibility to make sure that the trial is done ethically and in accordance with good clinical practices that are accepted worldwide.

The institutional review board is very important to ensure patient safety. Many institutions have their own institutional review board. There are some contract institutional review boards that different investigators can work with. The institutional review board reviews the study, looks at it primarily with patient safety in mind. They want to make sure that this is a reasonable, safe, ethical study for the patient to participate in. And they want to make sure the patient understands the risk. The institutional review board looks over the consent and really makes sure it is explaining to patient what exactly will happen in that study. So every investigator needs and IRB, institutional review board approval to start a study at their site. And then other things need to be reported to the IRB as well, if there's a problem, if there's an adverse event that's unexpected, that needs to go to the IRB so they can determine if it's safe to continue exposing patients to the study.

Clinical trials are very regimented and have very strict protocols for what happens. One of the most important criteria or things in the protocol are the entry and exclusion criteria for that trial. Clinical trials often want to answer a specific question in a specific patient population. And the inclusion criteria help define that patient population. It may be patients with a disease that have certain criteria based on how advanced they are, how many years they've had the disease, how sick they are, a number of different criteria like that. And then there's often exclusion criteria as well. We don't want patients who have too many other comorbidities or something that might limit their life or exposure to drug or obviously an important one, is they're competent to participate in the trial. So it may have exclusion criteria about cooperation, mental health issues, drug abuse, that sort of thing. So it's important to make sure that all the patients studied in the trial are similar to answer the question in this defined population.

The participation in a clinical trial is something that is decided upon by both the patient and the investigator. So remember, the patients need to willing participate. This is a choice that the patients have. They have to decide if they want to go into a clinical trial. And even once the patient decides that they're willing to go into the clinical trial, the investigator needs to make sure that they meet all the inclusion and exclusion criteria. So they both need to be on the same page.

It's very important for a patient to know all the options they have for their care before participating in a clinical trial. It needs to be a totally voluntary decision. It shouldn't change the care that you receive by your physician. So one thing that patients need to know is that this is ultimately their decision to participate. And then, of course, it's important to know the details about the trial. The responsibilities, the risks of the trial related medication or any trial related procedures.

There are many reasons a clinical trial can be successful or unsuccessful. It can be unsuccessful if the medication truly doesn't work and that's one of the reasons we do clinical trials. We want to understand if a medication works in a particular patient population. So that's an important reason that it might not be successful. Other reasons might be that it was designed poorly. A poor end point was chosen. There could be a number of different other issues.

Sometimes we don't know the results of clinical trials for a long time. A patient might be enrolled at a site early in the trial and it may take another year or two or three to finish enrolling the clinical trial. So sometimes it seems like they don't know the results but it may just be because the trial's not over. Often times the investigator can share the results of the trial with the patients once all is said and done.

There are many clinical trials for patients with diseases and there are other options for patients who don't have specific disease. Many clinical trials want to look at healthy controls to see the effect of a therapy in those patients as well. And then of course the Phase I trials often involve normal controls. Usually when a patient participates in a clinical trial it should not cost them any money and in fact all of the procedures that are specifically for the trial should be covered as part of the clinical trial.

Clinical trials are ways to learn about therapies. And sometimes we don't know the effects of the therapy so there are potentially situations where there might be side effects of a therapy or side effects of a procedure that the patient might consider is not the safest option for them and that's an important thing for the patient to consider before they go into a clinical trial. Remember by the time a patient is approached about a clinical trial, usually the FDA has signed off on for their study and of course the IRB has reviewed it and has assessed the risks of the study. Sometimes we take risks in order to get benefits as well. And this has to be balanced with patients, their diagnosis and how sick they are.

Clinical trials are so important because it's our way to learn about therapies that we want to get out to patients. For example, in the disease that I take care of, it's a rare disease, the very first therapy was approved the year I started practicing, 1995, that was the first time we had anything for patients. Now, I tell them about a dozen different options that they have to treat their disease. And they're doing so much better than they were in 1995. And we wouldn't have those therapies if it weren't for all of the patients who participated in clinical trials before that.

While clinical trials are voluntary for any patient it is important for a patient to consider doing good for their community and their disease state and how their participation in the clinical trial might help further knowledge and lead to therapies for patients like them down the road.

]]>fullEpisode 189 - Vallerie McLaughlin, MDVallerie McLaughlin, MD - Clinical Trials Pt. 2Thu, 13 Sep 2018 12:00:00 +0000Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center explains the basics of clinical trials in part 2 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Dr. McLaughlin is an editorial board member of Chest, a Guest Editor for the Journal of the American College of Cardiology, and past Editor-in-Chief of Advances in Pulmonary Hypertension.

]]>Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center explains the basics of clinical trials in part 2 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Dr. McLaughlin is an editorial board member of Chest, a Guest Editor for the Journal of the American College of Cardiology, and past Editor-in-Chief of Advances in Pulmonary Hypertension.

]]>11:18cleanblood,research,heart,awareness,therapies,asthma,breathless,rare,ph,trials,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdisease,clincalVallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center explains the basics of clinical trials in part 2 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Learn more about trials at www.phaware.global/clinicaltrials. #phaware285fullphaware global association Vallerie McLaughlin, MD - phaware® interview 188Tue, 11 Sep 2018 12:00:00 +0000

Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center breaks down the different phases of clinical trials in part 1 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Dr. McLaughlin is an editorial board member of Chest, a Guest Editor for the Journal of the American College of Cardiology, and past Editor-in-Chief of Advances in Pulmonary Hypertension.

Hi, I'm Vallerie McLaughlin, a cardiologist at the University of Michigan and my area of expertise is pulmonary hypertension.

Today, I'd like to talk to you about clinical trials.

A clinical trial is an investigation to answer a question as to whether a drug or device or some other maneuver works in a specific disease. Clinical trials are important and necessary to help further the therapies that we have available for many different diseases. The only way to get a drug or device or another intervention approved is to do a clinical trial to demonstrate that the drug works, and is safe for patients.

Clinical Trials can be divided into a number of different phases. Oftentimes, before you even hear about the Phase I clinical trial, there have been what we call preclinical testing, usually in animal models to look at the safety and the proof of concept of the mechanism of action of the drug. Once there is data, preclinical data, we often move into a phase one trial. A Phase I trial is usually a trial in healthy volunteers to get some information about the dosing of a drug, and about the side effects that might be expected with the drug.

A Phase II trial is often called a proof of concept trial. It's usually a smaller trial in patients with that disease, to get information about how effective the drug is to treat certain parameters of that disease. And again, safety is monitored all the way along. Usually, a drug proceeds into a Phase III trial if it's effective in Phase II. A Phase III trial is what we call a pivotal trial. It's usually a larger trial that has a clinically meaningful endpoint that the FDA will accept to approve a drug. And of course, safety is monitored throughout. A Phase III trial is what the sponsor goes to the FDA with to get approval for that agent. And then sometimes, there are Phase IV trials. The drug then has already been demonstrated to be effective in a certain disease, but we might want to study it in other diseases or at other points in the original disease, or with other drugs. And so, Phase IV trials can be lots of different scenarios.

Clinical trials have potential benefits, and they have potential risks for patients. Just by the very nature, a clinical trial is testing an agent that is unproven. And so, there could be side effects that have not yet been discovered that the patient may be exposed to. Sometimes, there's also risks of procedures that are done as part of a clinical trial, were measuring things during clinical trials. And so, patients may undergo additional procedures that they might not have undergone as part of the standard of care.

Many of our clinical trials are what we call placebo control. That's our way to tell if the drug is really effective compared to no drug it all. A placebo in a drug trial is often a sugar pill or an inactive pill that the patient takes, so that neither the patient nor the investigator knows if they're getting the active therapy. Clinical trials often are placebo controlled. So, some of the patients received the act of therapy and some of the patients receive the placebo. Patients are monitored throughout the clinical trial. And many clinical trials have safeguards in them so that if a patient receiving a placebo deteriorates, we can record that and offer the patient other therapy. Some trials also allow patients who were randomized to placebo in the placebo controlled portion to go into what's called an open label extension and get the active therapy after the placebo controlled portion of the trial.

Patient participation in clinical trials is always voluntary. We want to make sure the patient understands what they're getting themselves into before they go into the trial. In fact, many of what we call the consent forms for the patient or the subject are 10, 15, 20 pages long and describe the trial in detail. So, hopefully, the patient understands their commitment when they enter into the clinical trial. But again, clinical trial research is always voluntary. And if at some point the patient decides they no longer want to be in the trial, they should discuss that with the study team, and it's always their option to come out of the clinical trial.

Clinical trials are important for medicine in general, and sometimes patients in particular. There are many reasons a patient might volunteer for a clinical trial. Sometimes a patient might volunteer for a clinical trial because they have a serious disease and they have exhausted all of the currently FDA approved options, and a clinical trial might directly benefit them by giving them a therapy that's not yet commercially available. There are other patients who may not be in that situation, but may volunteer in a clinical trial for the greater good. Just think about it. All of our patients who are receiving therapies today are receiving those therapies because patients before them participated in clinical trials that led to the approval of those agents. So, some patients may be altruistic and even though they may not personally derive benefit from that clinical trial at that time, they know it could be the opportunity for a drug to be approved that would benefit a patient in the future.

So, open communication with a patient's physician and study team is really important as one considers clinical trial participation. In patients with rare diseases with limited options, it's important to ask about clinical trials at most appointments, so that you can learn what options there might be. If a patient decides they might be interested in participating in a clinical trial, it's very important to get as much information as you can about the trial. Much of the information is described in detail in the subject consent form. That should go over many issues, including the study visits, the procedures that might be done as part of a trial, what the patient's commitment is. It often involves more frequent visits and tests to the study site. It's important to also learn about the study medication, what the potential side effects are. By the time of trial gets into human studies, we know a little bit about the side effects of the medication. So, it's important to understand that.

Many physicians participate in clinical trials as investigators, particularly in rare diseases or complex diseases when we're always trying to learn. Some of the very experts that provide clinical care to patients also do clinical trials. And so, if oftentimes you can have the same doctor, sometimes that's considered a conflict and you have to see a different investigator on the study team. So, there may be circumstances in which either case can apply.

Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center breaks down the different phases of clinical trials in part 1 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Dr. McLaughlin is an editorial board member of Chest, a Guest Editor for the Journal of the American College of Cardiology, and past Editor-in-Chief of Advances in Pulmonary Hypertension.

Hi, I'm Vallerie McLaughlin, a cardiologist at the University of Michigan and my area of expertise is pulmonary hypertension.

Today, I'd like to talk to you about clinical trials.

A clinical trial is an investigation to answer a question as to whether a drug or device or some other maneuver works in a specific disease. Clinical trials are important and necessary to help further the therapies that we have available for many different diseases. The only way to get a drug or device or another intervention approved is to do a clinical trial to demonstrate that the drug works, and is safe for patients.

Clinical Trials can be divided into a number of different phases. Oftentimes, before you even hear about the Phase I clinical trial, there have been what we call preclinical testing, usually in animal models to look at the safety and the proof of concept of the mechanism of action of the drug. Once there is data, preclinical data, we often move into a phase one trial. A Phase I trial is usually a trial in healthy volunteers to get some information about the dosing of a drug, and about the side effects that might be expected with the drug.

A Phase II trial is often called a proof of concept trial. It's usually a smaller trial in patients with that disease, to get information about how effective the drug is to treat certain parameters of that disease. And again, safety is monitored all the way along. Usually, a drug proceeds into a Phase III trial if it's effective in Phase II. A Phase III trial is what we call a pivotal trial. It's usually a larger trial that has a clinically meaningful endpoint that the FDA will accept to approve a drug. And of course, safety is monitored throughout. A Phase III trial is what the sponsor goes to the FDA with to get approval for that agent. And then sometimes, there are Phase IV trials. The drug then has already been demonstrated to be effective in a certain disease, but we might want to study it in other diseases or at other points in the original disease, or with other drugs. And so, Phase IV trials can be lots of different scenarios.

Clinical trials have potential benefits, and they have potential risks for patients. Just by the very nature, a clinical trial is testing an agent that is unproven. And so, there could be side effects that have not yet been discovered that the patient may be exposed to. Sometimes, there's also risks of procedures that are done as part of a clinical trial, were measuring things during clinical trials. And so, patients may undergo additional procedures that they might not have undergone as part of the standard of care.

Many of our clinical trials are what we call placebo control. That's our way to tell if the drug is really effective compared to no drug it all. A placebo in a drug trial is often a sugar pill or an inactive pill that the patient takes, so that neither the patient nor the investigator knows if they're getting the active therapy. Clinical trials often are placebo controlled. So, some of the patients received the act of therapy and some of the patients receive the placebo. Patients are monitored throughout the clinical trial. And many clinical trials have safeguards in them so that if a patient receiving a placebo deteriorates, we can record that and offer the patient other therapy. Some trials also allow patients who were randomized to placebo in the placebo controlled portion to go into what's called an open label extension and get the active therapy after the placebo controlled portion of the trial.

Patient participation in clinical trials is always voluntary. We want to make sure the patient understands what they're getting themselves into before they go into the trial. In fact, many of what we call the consent forms for the patient or the subject are 10, 15, 20 pages long and describe the trial in detail. So, hopefully, the patient understands their commitment when they enter into the clinical trial. But again, clinical trial research is always voluntary. And if at some point the patient decides they no longer want to be in the trial, they should discuss that with the study team, and it's always their option to come out of the clinical trial.

Clinical trials are important for medicine in general, and sometimes patients in particular. There are many reasons a patient might volunteer for a clinical trial. Sometimes a patient might volunteer for a clinical trial because they have a serious disease and they have exhausted all of the currently FDA approved options, and a clinical trial might directly benefit them by giving them a therapy that's not yet commercially available. There are other patients who may not be in that situation, but may volunteer in a clinical trial for the greater good. Just think about it. All of our patients who are receiving therapies today are receiving those therapies because patients before them participated in clinical trials that led to the approval of those agents. So, some patients may be altruistic and even though they may not personally derive benefit from that clinical trial at that time, they know it could be the opportunity for a drug to be approved that would benefit a patient in the future.

So, open communication with a patient's physician and study team is really important as one considers clinical trial participation. In patients with rare diseases with limited options, it's important to ask about clinical trials at most appointments, so that you can learn what options there might be. If a patient decides they might be interested in participating in a clinical trial, it's very important to get as much information as you can about the trial. Much of the information is described in detail in the subject consent form. That should go over many issues, including the study visits, the procedures that might be done as part of a trial, what the patient's commitment is. It often involves more frequent visits and tests to the study site. It's important to also learn about the study medication, what the potential side effects are. By the time of trial gets into human studies, we know a little bit about the side effects of the medication. So, it's important to understand that.

Many physicians participate in clinical trials as investigators, particularly in rare diseases or complex diseases when we're always trying to learn. Some of the very experts that provide clinical care to patients also do clinical trials. And so, if oftentimes you can have the same doctor, sometimes that's considered a conflict and you have to see a different investigator on the study team. So, there may be circumstances in which either case can apply.

]]>fullEpisode 188 - Vallerie McLaughlin, MDVallerie McLaughlin, MD - Clinical Trials Pt. 1Mon, 10 Sep 2018 12:00:00 +0000Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center breaks down the different phases of clinical trials in part 1 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Dr. McLaughlin is an editorial board member of Chest, a Guest Editor for the Journal of the American College of Cardiology, and past Editor-in-Chief of Advances in Pulmonary Hypertension.

]]>Vallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center breaks down the different phases of clinical trials in part 1 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She is a Fellow of the American College of Cardiology, American College of Chest Physicians, and American Heart Association (AHA). Dr. McLaughlin has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Dr. McLaughlin is an editorial board member of Chest, a Guest Editor for the Journal of the American College of Cardiology, and past Editor-in-Chief of Advances in Pulmonary Hypertension.

]]>07:19cleanblood,research,heart,awareness,therapies,asthma,breathless,rare,ph,trials,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdisease,clincalVallerie McLaughlin, MD from Michigan Medicine's Cardiovascular Center breaks down the different phases of clinical trials in part 1 of her discussion. Dr. McLaughlin is the Director of the Pulmonary Hypertension Program at the University of Michigan in Ann Arbor. She has been the Principal Investigator of several major clinical trials of drug therapies for pulmonary arterial hypertension and has published numerous papers in this field. Learn more about trials at www.phaware.global/clinicaltrials. #phaware284fullphaware global associationBeth Decristofaro - phaware® interview 187Fri, 07 Sep 2018 12:00:00 +0000

Beth DeCristofaro is a pulmonary hypertension patient from Virginia. She discusses her diagnosis and involvement in clinical trial research. Beth is a married mother of two, and a former Hospice Chaplain.

I'm Beth Decristofaro, and I have idiopathic pulmonary arterial hypertension. I was diagnosed in October of 2017.

I actually was on vacation, had, looking back, what was a crisis because I didn't have many symptoms before that. In hindsight, I recognize now some of them, but I was functioning, working full-time. I was diagnosed after going to an ER short of breath, barely able to walk, feeling horrible, and the ER doctor said, "We've called in the cardiologist because we have no idea what's going on with you." That weekend, I was diagnosed. Within about four weeks, I was on an IV and an oral med. I had originally been put on two orals, but did not respond well, and so my PH specialist said, "The IV is the way to go." I've been on that since and responded very well, so they were right.

I was diagnosed really quickly, and I know that's unusual for this disease. It came out of the blue in many ways. I had noticed about six months before that I was having trouble walking up the stairs, but I figured I was out of shape. So, I did not even really understand when I was first told what it was. I think I just knew that this was pretty serious, and so what do we do next? It was later when I got to an excellent PH specialist cardiologist that it began to sink in. I had been in pretty severe heart failure. Then it began to hit me like a ton of bricks. What does this mean? Do I, in fact, have a real life-limited type of a diagnosis now? My family and I kind of began to look at that and say, "What do we need to do?"

What was interesting is as I was beginning to understand and realize the limitations, I began to also respond quickly to the medicines. It was always a, oh my goodness, I'm living every day with my mortality in a way I had never done before, but on the other hand, I knew I was being helped, and so it was sort of this astounding off balance feeling for a very, very long time. I was put on the IV therapy in end of October, and I think it was probably the end of March, beginning of April, that my own doctor said, "Hey, would you be interested at all in being part of a clinical trial?" Then it was April and May that I got involved in it. It's been better. I've had a better response for me, personally, than I ever expected, which is just amazing.

My background has been in healthcare for many years, so I was always open to that idea, but didn't really realize you could do that as an active patient. I am on a trial that is a small phase one trial, and it's called ABI-009 for Patients with Severe Pulmonary Arterial Hypertension. It is an interesting trial because this drug is already FDA approved. It's an immunosuppressant that's been used for years for transplantation patients. They're looking at what immunosuppressant properties might also be appropriate for PAH. They are finding certain similarities, and this also has to do with inflammation in the arteries, so it's a very small trial. There's no placebo. We all know we're getting this particular drug. I think it's really sort of sped up my positive response. I'm seeing a big difference in my own health.

My overall feeling about clinical trials is that they're absolutely necessary. I had never bothered looking into one very closely. When I received the information, and they give you copious amounts of information and spend a lot of time explaining the process and answering questions, I did really want to hear what the risks were versus the potential outcomes. I was willing to settle for a neutral outcome for me, quite honest, but I also didn't want to put my own health at risk, mainly thinking of my family and my responsibilities. In this case, I felt that the risks absolutely were not so overwhelming. It was worth doing.

And, another big factor for me is not only is my own doctor one of the researchers in this trial, but I live close enough that going once a week, or even multiple times a week, because that has happened, too, was not completely out of possibility for me. In fact, it was a little bit of reward in that I did have to quit my job, due to the PH, and so here I could give back a little bit in a way that I hadn't been able to do since I was no longer working. So, it didn't take a lot for me, but I did research it pretty thoroughly to say, "Absolutely, let's go ahead and let's do this."

This particular trial I'm on is 17 weeks long, which counts pre-tests and post-tests. One of the advantages, in my opinion, is you get to see a doctor every single week. I have had an echo done every four weeks and lab works consistently. In order to start, I did have to have a right heart catheterization and will have one at the end, but that's okay for me if they're showing me what my pressures are doing. Lots of blood sticks, but that's okay. We can handle those. There is a stipend that I get every time I go in and that helps defray costs, parking and what have you.

I will be finishing up now in three weeks. Again, that battery of tests is the very final week, and then I'll be able to also get that wrap up summary from the researcher, who is my own doctor. And so again, I find that to be a real plus for anyone who is able and willing to do clinical trials, is the close monitoring, not only of what's happening positive, but absolutely if there's any side effects or concerns, they ask you about it and monitor those. Any time you want to back out, you can do that, too, but I have not been tempted in the least.

If you're considering thinking about or wondering about clinical trials, I would say start with your own doctor. What does he or she know about it? And help you begin to think about what might be in your best interest, but also what are promising avenues, and then look carefully. Look carefully at the risks, but also look at the benefits. I, personally, have had no complaints and feel like it's a chance to do a little something for the people coming after me, because frankly, I'm where I am doing so well because of the people who came before me.

As you consider, of course, there is the federal website, but also phaware global association now has a matching tool (www.phaware.global/clinicaltrials), that you can plug in where you are and what are some specifics. There are questions that you answer about your own PH situation. Trials will come up in your area that you are probably eligible for, and you can even do some research right on there. Does anything look interesting? Anything look promising? That kind of easy way to get in the process is something you could do today.

Beth DeCristofaro is a pulmonary hypertension patient from Virginia. She discusses her diagnosis and involvement in clinical trial research. Beth is a married mother of two, and a former Hospice Chaplain.

I'm Beth Decristofaro, and I have idiopathic pulmonary arterial hypertension. I was diagnosed in October of 2017.

I actually was on vacation, had, looking back, what was a crisis because I didn't have many symptoms before that. In hindsight, I recognize now some of them, but I was functioning, working full-time. I was diagnosed after going to an ER short of breath, barely able to walk, feeling horrible, and the ER doctor said, "We've called in the cardiologist because we have no idea what's going on with you." That weekend, I was diagnosed. Within about four weeks, I was on an IV and an oral med. I had originally been put on two orals, but did not respond well, and so my PH specialist said, "The IV is the way to go." I've been on that since and responded very well, so they were right.

I was diagnosed really quickly, and I know that's unusual for this disease. It came out of the blue in many ways. I had noticed about six months before that I was having trouble walking up the stairs, but I figured I was out of shape. So, I did not even really understand when I was first told what it was. I think I just knew that this was pretty serious, and so what do we do next? It was later when I got to an excellent PH specialist cardiologist that it began to sink in. I had been in pretty severe heart failure. Then it began to hit me like a ton of bricks. What does this mean? Do I, in fact, have a real life-limited type of a diagnosis now? My family and I kind of began to look at that and say, "What do we need to do?"

What was interesting is as I was beginning to understand and realize the limitations, I began to also respond quickly to the medicines. It was always a, oh my goodness, I'm living every day with my mortality in a way I had never done before, but on the other hand, I knew I was being helped, and so it was sort of this astounding off balance feeling for a very, very long time. I was put on the IV therapy in end of October, and I think it was probably the end of March, beginning of April, that my own doctor said, "Hey, would you be interested at all in being part of a clinical trial?" Then it was April and May that I got involved in it. It's been better. I've had a better response for me, personally, than I ever expected, which is just amazing.

My background has been in healthcare for many years, so I was always open to that idea, but didn't really realize you could do that as an active patient. I am on a trial that is a small phase one trial, and it's called ABI-009 for Patients with Severe Pulmonary Arterial Hypertension. It is an interesting trial because this drug is already FDA approved. It's an immunosuppressant that's been used for years for transplantation patients. They're looking at what immunosuppressant properties might also be appropriate for PAH. They are finding certain similarities, and this also has to do with inflammation in the arteries, so it's a very small trial. There's no placebo. We all know we're getting this particular drug. I think it's really sort of sped up my positive response. I'm seeing a big difference in my own health.

My overall feeling about clinical trials is that they're absolutely necessary. I had never bothered looking into one very closely. When I received the information, and they give you copious amounts of information and spend a lot of time explaining the process and answering questions, I did really want to hear what the risks were versus the potential outcomes. I was willing to settle for a neutral outcome for me, quite honest, but I also didn't want to put my own health at risk, mainly thinking of my family and my responsibilities. In this case, I felt that the risks absolutely were not so overwhelming. It was worth doing.

And, another big factor for me is not only is my own doctor one of the researchers in this trial, but I live close enough that going once a week, or even multiple times a week, because that has happened, too, was not completely out of possibility for me. In fact, it was a little bit of reward in that I did have to quit my job, due to the PH, and so here I could give back a little bit in a way that I hadn't been able to do since I was no longer working. So, it didn't take a lot for me, but I did research it pretty thoroughly to say, "Absolutely, let's go ahead and let's do this."

This particular trial I'm on is 17 weeks long, which counts pre-tests and post-tests. One of the advantages, in my opinion, is you get to see a doctor every single week. I have had an echo done every four weeks and lab works consistently. In order to start, I did have to have a right heart catheterization and will have one at the end, but that's okay for me if they're showing me what my pressures are doing. Lots of blood sticks, but that's okay. We can handle those. There is a stipend that I get every time I go in and that helps defray costs, parking and what have you.

I will be finishing up now in three weeks. Again, that battery of tests is the very final week, and then I'll be able to also get that wrap up summary from the researcher, who is my own doctor. And so again, I find that to be a real plus for anyone who is able and willing to do clinical trials, is the close monitoring, not only of what's happening positive, but absolutely if there's any side effects or concerns, they ask you about it and monitor those. Any time you want to back out, you can do that, too, but I have not been tempted in the least.

If you're considering thinking about or wondering about clinical trials, I would say start with your own doctor. What does he or she know about it? And help you begin to think about what might be in your best interest, but also what are promising avenues, and then look carefully. Look carefully at the risks, but also look at the benefits. I, personally, have had no complaints and feel like it's a chance to do a little something for the people coming after me, because frankly, I'm where I am doing so well because of the people who came before me.

As you consider, of course, there is the federal website, but also phaware global association now has a matching tool (www.phaware.global/clinicaltrials), that you can plug in where you are and what are some specifics. There are questions that you answer about your own PH situation. Trials will come up in your area that you are probably eligible for, and you can even do some research right on there. Does anything look interesting? Anything look promising? That kind of easy way to get in the process is something you could do today.

]]>Beth DeCristofaro is a pulmonary hypertension patient from Virginia. She discusses her diagnosis and involvement in clinical trial research. Beth is a married mother of two, and a former Hospice Chaplain.

]]>08:02cleanblood,research,heart,awareness,therapies,asthma,breathless,rare,ph,trials,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdisease,clincalBeth DeCristofaro is a pulmonary hypertension patient from Virginia. She discusses her diagnosis and involvement in clinical trial research. Beth is a married mother of two, and a former Hospice Chaplain.
Learn more about pulmonary hypertension trials at www.phaware.global/clinicaltrials. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware 283fullphaware global associationAlex Flipse - phaware® interview 186Tue, 04 Sep 2018 12:00:00 +0000

Alex Flipse was diagnosed with pulmonary hypertension on September 3, 1998. On the 20th anniversary of her diagnosis, Alex details her journey battling both depression and PH.

My name is Alex Flipse. I have pulmonary arterial hypertension. I have pulmonary venous hypertension, and I am in Southern California in Riverside County.

I had just delivered my third, and final, baby. Probably three months after her birth, I started feeling really, really exhausted all the time. I attributed that to the depression that I've had for all my life, basically and, of course, just having a brand new baby, as well. Things started going down the road and I would forget about how tired I was always feeling. Actually, for quite a while I would sleep like maybe 20 hours a day. I would get up in the morning and feed the children and put them in front of the TV and fall asleep on the couch. I'd get up in time to make them some lunch, put them in front of the TV and fall asleep on the couch. I'd get up in time to make dinner. My ex would come home. I'd say, "Here are the kids. I'm exhausted," and I'd be asleep for the rest of the night. That's how my PH journey began.

I was misdiagnosed along the way, but I do believe that was my own fault, because I kept saying, "I'm always tired. I'm short of breath. I'm always this." It would always be, "But, I have depression. We just moved from here to here, and I left my family in California, and that's really making me depressed," until one time I started passing out. I would go back to the same doctor, who was the family doctor. There was a time when my sister, Debbie, was visiting us during her summer months from University. For some reason, I got it in my head I had to take out the garbage. It was in the middle of the day. I know the kids were in school and I said, "I just got to go do this," so I jumped up and I took it out. I came back inside and I went down. I just completely passed out. On my way down, thankfully, she caught me. She helped me come back and then she went and got my ex. He took me to go see my primary care doctor.

Thankfully, my primary care doctor wasn't there. Somebody else was there, you know on-call doctor, and he listened to me and he said, "This is not in your head. This is in your heart," and immediately sent me in for an echo. The next day I went and saw the cardiologist and he said, "We think you have something called pulmonary hypertension," and that was it. This was September 3, 1998. When I went in to see the specialist over in Houston, she had me get a right heart catheterization. She did it herself. When I woke up from that right heart catheterization, that they put me under, I was on IV therapy. It took me four years to be able to actually get out of bed. I was Stage IV when I was diagnosed, but more than that, my depression had taken over, and I could not get out of bed, I could not take care of my kids. I could not do anything. My kids' father was stationed something like six hours away, so he would only maybe only come on his weekends, which was usually only once a month.

My mother ended up moving out to Texas with us to help me with the children. But, during that entire time I was overcome. I couldn't get out of bed, I couldn't wake up, I couldn't anything. It was difficult for me. It was difficult for my children, who have all suffered because of it. None of them, thank goodness, blame me for my condition, but they do blame me for not being there for them when they were younger.

The oral pill was approved by the FDA. As soon as it was introduced into the FDA, then I was able to slowly get back to life. I was able to slowly do more and be there for the kids more, but it did take quite some time. My oldest actually had to grow up really fast and take care of her sisters, and change diapers, and everything else. My youngest was two years old when I was diagnosed. It was just really, really hard on all of us. For my children I was able to set things up so that they could help themselves. I got things that they could eat themselves, or they could prepare themselves. I filled the bottom shelves of the refrigerator with snacks that they could get to. I had baskets of coloring books, and toys, and things in my bedroom so that they could sit with me in bed when I was up, and that sort of thing, and then, I could help them with their homework, or whatever.

After I got off of the IV therapy and started onto the oral, I started searching online for information and for help and I found several different websites, lots and lots of people who were in my same condition as far as pulmonary hypertension. I started finding ways to combat the depression and work with the pulmonary hypertension, not combat it but to work with it, and I did that slowly and slowly the depression started getting better and better. But as depression does, it sneaks up rather quickly, and for me it never left me. I still have it every single day. I wake up with it and I go to sleep with it, and it's always there in the back of my head regardless of how bad it is or how good it is.

I always make sure that I have people to contact when I get really low. I always be sure to have music, or movies, or things that I can avert my brain to, so that I'm not constantly only thinking inward. I have journals that I write into, and it's a matter of actually taking pen to paper, because if you type it out ... There's some sort of science to it. I understand what it is, and I learned this actually when I was inpatient in mental hospitals. There should be absolutely no stigma with that, because mental health, depression, anxiety, all of that is a disease as much as PH is. So, going into the hospital to take care of my mental health, I've done that many times over the years. If you need it, please do it. Dealing with the depression and dealing with all the anxiety that comes with pulmonary hypertension more than the depression ... For many, many years, and thank god I'm over it now, but it creeps up to me every now and again, but for many, many years I have always thought to myself, "I can't make plans so far into the future, because who knows if I'm still going to be here." It took me a long time to realize, "I will still be here. I will still kick PH in the butt, and I will still be able to thrive and succeed."

When I got sick I always wanted to know why did I get sick? Why did God choose me for this? Why, why, why, why? It started to dawn on me, 2009, I suppose, maybe 10 years after diagnosis, I have to be able to help people. My way of dealing is by helping. My way of helping is by doing. I don't have the funds to be able to donate left and right to all the organizations that need it, and I wish that I could. My way to help, since I can't give, is to do for people, and that sort of thing. That has helped me tremendously.

I started my very first support group in Texas. I think it might have been six years after diagnosis. I was on the oral medication already. I did it because I was very lonely. There was not a soul that I knew in this little town that I lived in that had pulmonary hypertension. I came to find out there was a young woman there who had PH and, unfortunately, she passed away a few years after I met her. But, I started the support group down there, and there were people coming from hundreds of miles away, and that feeling of helping people was tremendous for me. It wasn't only therapeutic, it was also a sense of being able to complete something, being productive, being helpful. It is a huge relief for me to be able to do that, and it was like this is the gift that I've been given. I've been given life after the two years that they told me I was only going to live, and I have to do something with this time. Even though I can't work, and I can't give, and I can't whatever, these are the things that I can do.

On September 3, I was given my official diagnosis of pulmonary hypertension by my specialist in Houston in 1998. That's been 20 years now. I never thought that I was going to live past the two years they told me I was going to have. I never thought I'd get to see my children grow up, and now I have grandchildren. There's so much I would love to do. There is so much I would love to see. I would love to travel the world and see things and everything else. I like to live vicariously, as they say, through my kids.

My youngest daughter, I am so, so proud of her. She graduated from the University of Texas A&M. She's gone skydiving. She's gone on a hot air balloon ride. She rides horses on the beach. She deep sea fishes. She does all of these things, and all of these things I wish that I could do. They're all on my bucket list. So, I see everything that she does and I would love to be able to do all that. I want to continue helping the community. I want to be able to maybe give a little bit of advice to the newbies, to the newly diagnosed, and show them that there is life after diagnosis. There is life after the two years that they give us, and I wish they would stop doing that, but there definitely is life after diagnosis.

If I could give a piece of advice to a newly diagnosed patient it would be, "Take your meds like you're supposed to, find a support group, go to www.phaware.global, go online to Facebook and find thousands of PH friends, because we're all over the place. Know that you are never alone, know that life is not over. Even though pulmonary hypertension will change how you live, it will not end your life, it will not end how you live, you just have to change and be willing to accept your new life and your new normal. If you want, wherever you are in the world you can find me online @flippymom. I'm @flippymom on Facebook, on Instagram, Snapchat, allover.

Alex Flipse was diagnosed with pulmonary hypertension on September 3, 1998. On the 20th anniversary of her diagnosis, Alex details her journey battling both depression and PH.

My name is Alex Flipse. I have pulmonary arterial hypertension. I have pulmonary venous hypertension, and I am in Southern California in Riverside County.

I had just delivered my third, and final, baby. Probably three months after her birth, I started feeling really, really exhausted all the time. I attributed that to the depression that I've had for all my life, basically and, of course, just having a brand new baby, as well. Things started going down the road and I would forget about how tired I was always feeling. Actually, for quite a while I would sleep like maybe 20 hours a day. I would get up in the morning and feed the children and put them in front of the TV and fall asleep on the couch. I'd get up in time to make them some lunch, put them in front of the TV and fall asleep on the couch. I'd get up in time to make dinner. My ex would come home. I'd say, "Here are the kids. I'm exhausted," and I'd be asleep for the rest of the night. That's how my PH journey began.

I was misdiagnosed along the way, but I do believe that was my own fault, because I kept saying, "I'm always tired. I'm short of breath. I'm always this." It would always be, "But, I have depression. We just moved from here to here, and I left my family in California, and that's really making me depressed," until one time I started passing out. I would go back to the same doctor, who was the family doctor. There was a time when my sister, Debbie, was visiting us during her summer months from University. For some reason, I got it in my head I had to take out the garbage. It was in the middle of the day. I know the kids were in school and I said, "I just got to go do this," so I jumped up and I took it out. I came back inside and I went down. I just completely passed out. On my way down, thankfully, she caught me. She helped me come back and then she went and got my ex. He took me to go see my primary care doctor.

Thankfully, my primary care doctor wasn't there. Somebody else was there, you know on-call doctor, and he listened to me and he said, "This is not in your head. This is in your heart," and immediately sent me in for an echo. The next day I went and saw the cardiologist and he said, "We think you have something called pulmonary hypertension," and that was it. This was September 3, 1998. When I went in to see the specialist over in Houston, she had me get a right heart catheterization. She did it herself. When I woke up from that right heart catheterization, that they put me under, I was on IV therapy. It took me four years to be able to actually get out of bed. I was Stage IV when I was diagnosed, but more than that, my depression had taken over, and I could not get out of bed, I could not take care of my kids. I could not do anything. My kids' father was stationed something like six hours away, so he would only maybe only come on his weekends, which was usually only once a month.

My mother ended up moving out to Texas with us to help me with the children. But, during that entire time I was overcome. I couldn't get out of bed, I couldn't wake up, I couldn't anything. It was difficult for me. It was difficult for my children, who have all suffered because of it. None of them, thank goodness, blame me for my condition, but they do blame me for not being there for them when they were younger.

The oral pill was approved by the FDA. As soon as it was introduced into the FDA, then I was able to slowly get back to life. I was able to slowly do more and be there for the kids more, but it did take quite some time. My oldest actually had to grow up really fast and take care of her sisters, and change diapers, and everything else. My youngest was two years old when I was diagnosed. It was just really, really hard on all of us. For my children I was able to set things up so that they could help themselves. I got things that they could eat themselves, or they could prepare themselves. I filled the bottom shelves of the refrigerator with snacks that they could get to. I had baskets of coloring books, and toys, and things in my bedroom so that they could sit with me in bed when I was up, and that sort of thing, and then, I could help them with their homework, or whatever.

After I got off of the IV therapy and started onto the oral, I started searching online for information and for help and I found several different websites, lots and lots of people who were in my same condition as far as pulmonary hypertension. I started finding ways to combat the depression and work with the pulmonary hypertension, not combat it but to work with it, and I did that slowly and slowly the depression started getting better and better. But as depression does, it sneaks up rather quickly, and for me it never left me. I still have it every single day. I wake up with it and I go to sleep with it, and it's always there in the back of my head regardless of how bad it is or how good it is.

I always make sure that I have people to contact when I get really low. I always be sure to have music, or movies, or things that I can avert my brain to, so that I'm not constantly only thinking inward. I have journals that I write into, and it's a matter of actually taking pen to paper, because if you type it out ... There's some sort of science to it. I understand what it is, and I learned this actually when I was inpatient in mental hospitals. There should be absolutely no stigma with that, because mental health, depression, anxiety, all of that is a disease as much as PH is. So, going into the hospital to take care of my mental health, I've done that many times over the years. If you need it, please do it. Dealing with the depression and dealing with all the anxiety that comes with pulmonary hypertension more than the depression ... For many, many years, and thank god I'm over it now, but it creeps up to me every now and again, but for many, many years I have always thought to myself, "I can't make plans so far into the future, because who knows if I'm still going to be here." It took me a long time to realize, "I will still be here. I will still kick PH in the butt, and I will still be able to thrive and succeed."

When I got sick I always wanted to know why did I get sick? Why did God choose me for this? Why, why, why, why? It started to dawn on me, 2009, I suppose, maybe 10 years after diagnosis, I have to be able to help people. My way of dealing is by helping. My way of helping is by doing. I don't have the funds to be able to donate left and right to all the organizations that need it, and I wish that I could. My way to help, since I can't give, is to do for people, and that sort of thing. That has helped me tremendously.

I started my very first support group in Texas. I think it might have been six years after diagnosis. I was on the oral medication already. I did it because I was very lonely. There was not a soul that I knew in this little town that I lived in that had pulmonary hypertension. I came to find out there was a young woman there who had PH and, unfortunately, she passed away a few years after I met her. But, I started the support group down there, and there were people coming from hundreds of miles away, and that feeling of helping people was tremendous for me. It wasn't only therapeutic, it was also a sense of being able to complete something, being productive, being helpful. It is a huge relief for me to be able to do that, and it was like this is the gift that I've been given. I've been given life after the two years that they told me I was only going to live, and I have to do something with this time. Even though I can't work, and I can't give, and I can't whatever, these are the things that I can do.

On September 3, I was given my official diagnosis of pulmonary hypertension by my specialist in Houston in 1998. That's been 20 years now. I never thought that I was going to live past the two years they told me I was going to have. I never thought I'd get to see my children grow up, and now I have grandchildren. There's so much I would love to do. There is so much I would love to see. I would love to travel the world and see things and everything else. I like to live vicariously, as they say, through my kids.

My youngest daughter, I am so, so proud of her. She graduated from the University of Texas A&M. She's gone skydiving. She's gone on a hot air balloon ride. She rides horses on the beach. She deep sea fishes. She does all of these things, and all of these things I wish that I could do. They're all on my bucket list. So, I see everything that she does and I would love to be able to do all that. I want to continue helping the community. I want to be able to maybe give a little bit of advice to the newbies, to the newly diagnosed, and show them that there is life after diagnosis. There is life after the two years that they give us, and I wish they would stop doing that, but there definitely is life after diagnosis.

If I could give a piece of advice to a newly diagnosed patient it would be, "Take your meds like you're supposed to, find a support group, go to www.phaware.global, go online to Facebook and find thousands of PH friends, because we're all over the place. Know that you are never alone, know that life is not over. Even though pulmonary hypertension will change how you live, it will not end your life, it will not end how you live, you just have to change and be willing to accept your new life and your new normal. If you want, wherever you are in the world you can find me online @flippymom. I'm @flippymom on Facebook, on Instagram, Snapchat, allover.

]]>10:08cleanblood,research,heart,awareness,therapies,depression,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pah,pulmonaryhypertension,phaware,lungdiseaseAlex Flipse was diagnosed with pulmonary hypertension on September 3, 1998. On the 20th anniversary of her diagnosis, Alex details her journey battling both depression and PH.
Learn more about pulmonary hypertension at www.phaware365.global. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware @flippymom282fullphaware global associationDunbar Ivy, MD - phaware® interview 185Fri, 31 Aug 2018 12:00:00 +0000

Dr. Dunbar Ivy discusses the work of the Pediatric Pulmonary Hypertension Network, (PPHNet) on the network's 10th anniversary. PPHNet is a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease.

Hi, this is Dunbar Ivy. I'm Chief of Pediatric Cardiology at the University of Colorado School of Medicine and Director of the Pediatric Pulmonary Hypertension Program at Children's Hospital of Colorado.

I'd like to talk to you about the Pediatric Pulmonary Hypertension Network [PPHNet]. It's our tenth birthday. When we first started the network, we were a loosely associated group who had strong interest in pulmonary hypertension. Our initial goals were to learn how to treat patients better and to develop guidelines.

Over the last 10 years, we've been successful in some areas and challenged in others. I think our successes have been in the development of some guidelines, particularly for pediatric pulmonary hypertension in general and also for children with bronchopulmonary dysplasia, diaphragmatic hernia, and treatment of pulmonary hypertension in a group of patients with prematurity and pulmonary hypertension. Our group has been very involved in the World Symposium of Pulmonary Hypertension in driving recognition of pediatric causes of pulmonary hypertension in the classification of pulmonary hypertension and to our adult colleagues, because as we get better at treating children they're going to become and are becoming adults, and our adult colleagues need to know and understand these unique causes, these developmental causes of pulmonary hypertension.

I think the next frontier is helping the regulatory agencies and industry to define how to study these medicines in children to the benefit of the physicians, the families, the regulatory agencies, so that we can do a better job of knowing how to use medications in children. We've been very fortunate to have an extremely collaborative group and the senior members of the group have very much felt that we need to support our junior colleagues in their professional careers. That makes for a collegial environment. When we have an idea that comes up, then there's a discussion about who wants to take the lead and people are very supportive of spreading out the ideas. An example would be a genetic abnormality called TBX4 that was initially discovered to be strongly associated with children with pulmonary hypertension in the Netherlands and our group has taken that and then people have contributed their cases where they found this. We found that it's a cause of persistent pulmonary hypertension of the newborn, so this helps clinicians try to figure out why a baby may have PPHN. There is no direct targeted therapy for that, but I think it's an example of how the group has worked together for the common good.

I think the next frontier is to expand the group. There are more and more people with a dedicated interest in pulmonary hypertension and this will allow for better treatment of children across the United States and Canada because it's hard to travel. I think the network will expand and that will allow us to enroll more patients in our registry and then have more expert care throughout the country. That will, I think, be an important goal.

Dr. Dunbar Ivy discusses the work of the Pediatric Pulmonary Hypertension Network, (PPHNet) on the network's 10th anniversary. PPHNet is a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease.

Hi, this is Dunbar Ivy. I'm Chief of Pediatric Cardiology at the University of Colorado School of Medicine and Director of the Pediatric Pulmonary Hypertension Program at Children's Hospital of Colorado.

I'd like to talk to you about the Pediatric Pulmonary Hypertension Network [PPHNet]. It's our tenth birthday. When we first started the network, we were a loosely associated group who had strong interest in pulmonary hypertension. Our initial goals were to learn how to treat patients better and to develop guidelines.

Over the last 10 years, we've been successful in some areas and challenged in others. I think our successes have been in the development of some guidelines, particularly for pediatric pulmonary hypertension in general and also for children with bronchopulmonary dysplasia, diaphragmatic hernia, and treatment of pulmonary hypertension in a group of patients with prematurity and pulmonary hypertension. Our group has been very involved in the World Symposium of Pulmonary Hypertension in driving recognition of pediatric causes of pulmonary hypertension in the classification of pulmonary hypertension and to our adult colleagues, because as we get better at treating children they're going to become and are becoming adults, and our adult colleagues need to know and understand these unique causes, these developmental causes of pulmonary hypertension.

I think the next frontier is helping the regulatory agencies and industry to define how to study these medicines in children to the benefit of the physicians, the families, the regulatory agencies, so that we can do a better job of knowing how to use medications in children. We've been very fortunate to have an extremely collaborative group and the senior members of the group have very much felt that we need to support our junior colleagues in their professional careers. That makes for a collegial environment. When we have an idea that comes up, then there's a discussion about who wants to take the lead and people are very supportive of spreading out the ideas. An example would be a genetic abnormality called TBX4 that was initially discovered to be strongly associated with children with pulmonary hypertension in the Netherlands and our group has taken that and then people have contributed their cases where they found this. We found that it's a cause of persistent pulmonary hypertension of the newborn, so this helps clinicians try to figure out why a baby may have PPHN. There is no direct targeted therapy for that, but I think it's an example of how the group has worked together for the common good.

I think the next frontier is to expand the group. There are more and more people with a dedicated interest in pulmonary hypertension and this will allow for better treatment of children across the United States and Canada because it's hard to travel. I think the network will expand and that will allow us to enroll more patients in our registry and then have more expert care throughout the country. That will, I think, be an important goal.

]]>Dr. Dunbar Ivy discusses the work of the Pediatric Pulmonary Hypertension Network, (PPHNet) on the network's 10th anniversary. PPHNet is a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease.

]]>03:43cleannovel,blood,research,heart,pediatric,awareness,therapies,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseaseDr. Dunbar Ivy discusses the work of the Pediatric Pulmonary Hypertension Network, (PPHNet) on the network's 10th anniversary. PPHNet is a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease.
Learn more about pulmonary hypertension at www.phaware365.global. Follow us @phaware. Engage for a cure: www.phaware.global/donate #phaware #phawareMD281fullphaware global associationRoberta Keller, MD - phaware® interview 184Tue, 28 Aug 2018 12:00:00 +0000

Dr. Roberta Keller discusses the work of the Pediatric Pulmonary Hypertension Network, (PPHNet) on the network's 10th anniversary. PPHNet is a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease.

My name is Dr. Roberta Keller. I'm a professor of pediatrics at the University of California San Francisco Benioff Children's Hospital here in San Francisco. I'm a neonatologist, and my primary clinical and research interests is in the evaluation and management of newborns and children with cardiopulmonary disorders.

Today, I'd like to talk to you a little bit about the Pediatric Pulmonary Hypertension Network, or PPHNet.

We are a group of ten centers now, which was originally formed about 10 years ago. We're just celebrating our 10th anniversary. It was really the brainchild of Dr. Steve Abman at the University of Colorado and Colorado Children's Hospital in Denver. He saw a real need to bring together people who took care of children with pulmonary vascular disease and pulmonary hypertension and really tried to combine our experience and our efforts toward learning as much as possible about evaluating and treating children with pulmonary vascular disease and pulmonary hypertension.

So PPHNet originally started with eight centers. UCSF was one of those original centers. One of the things that's really great about PPHNet is that it represents people who care for these children from all different disciplines. Of course, there are pediatric cardiologists, which have been the mainstay of caring for children with pulmonary vascular disease, but also pediatric pulmonologists, pediatric critical care physicians, and neonatologists and really all working together toward the common goal of improving our understanding and treatment of affected children.

One of the great things about that is that we all come from different perspectives and have different ways of thinking about the problems that the children present. Of course, one of the challenges is that we aren't always going to agree on how to approach things or what to focus on specifically. We have some very, very lively discussions around how to focus our efforts. I think really it's been very, very productive because it makes us think about things in a different way, consider other perspectives, and really try to bring all of those view together toward improving the care of these patients through our work.

I think one of the things that's really unique about pediatrics is that we really consider growth and development in all of the decisions we make about children in terms of the state of their disease or condition and whether they could potentially benefit from additional therapies or whether we can potentially start to withdraw therapies over time.

In the adult pulmonary hypertension community and adult cardiovascular community, functional class has always been an important way of thinking about how people function in the setting of their disorder or their condition. If you look at the traditional or standard functional class assessments, those are pretty difficult to apply to certainly younger children, as they have a lot to with people's activities of daily living and when they become short of breath.

Honestly, in children, sometimes that's not even possible to get that history from somebody, whether or not they're short of breath. Their activities of daily living are very different over time. We think about a six-month-old versus a one-year-old versus a three-year-old, that is a constantly changing target. We really like to think about functional class in a way that is more specific to children who are growing and developing and incorporating both developmental milestones as well as nutrition and growth and what's needed for them to have adequate nutrition, and of course finally, whether or not they're able to stay in school and how they function in that environment.

The Pulmonary Vascular Research Institute, or PVRI, several years ago in 2011 published a proposed pediatric functional class, which has really helped us with thinking about these children in terms of their status over time and whether they are improving with their therapies or whether they're stalling out or actually worsening. That's been incredibly helpful.

I think that there's other ways of doing this without formally assigning a pediatric functional class. Those are of course are important, but it really incorporates the things we think about, for instance, related to growth. Are children growing well? Are they not growing well? Somebody who might seem to have stable disease, or even mildly improving disease, by imaging or by cardiac catheterization, may in fact not be growing well. So we decide not to withdraw therapies that we might otherwise withdraw in somebody who had those improving metrics. Similarly, a child who seems to have stable numbers but who is falling off in their growth or development or missing more school, we might decide to escalate therapies in order to try and improve their functional status.

One of the things that we worked really hard on, and I think has been covered before in this podcast, are the guidelines of pediatric pulmonary hypertension. Those guidelines are really a slimmed down version of all the work that we did to try and evaluate all the different aspects of pulmonary vascular disease and the evaluation and management of children with that condition.

I think if you look at the literature now, we see that many people are citing those guidelines, many people are reading those guidelines, and they're really contributing to initiation of evaluation of patients prior to them being referred to comprehensive centers and I hope really contributing to the understanding that many practitioners have of the pulmonary vascular disease and how it contributes to the conditions that the children may have as a lot of pulmonary vascular disease and pulmonary hypertension in pediatrics is actually secondary to other conditions, which is unique I think from the adult population.

We've also looked at a number of these conditions more in-depth and published some papers to help the overall community guide their initial approach to those kinds of patients and also identify what the gaps in knowledge are and what we need to really think about going forward.

Now, in PPHNet, we have been working on a registry. We have almost 1,500 children with pulmonary vascular disease in that registry consented for their information to be included. We're now able to look at those data, of course without any identifying identifiers, but in aggregate, to try and understand how these conditions are presenting variably in different children at different ages and what types of therapies we are using and whether or not we can tell if those therapies are efficacious.

In addition, I think what we see in children in general is that many conditions can actually improve over time with organ growth or development. I think we may be seeing that in the pediatric population that we don't just see the clinical improvement over time or functional improvement over time, but maybe sometimes even resolution because those children are growing new lung tissue and new vascular tissue and really able to improve vastly clinically and come off of their medications.

Now, whether those will be long-term successes or whether they will need to at some point go back on medications, we don't know yet. Obviously, we're all anxious about that and continuing to follow those children very closely. But I think that now with that large registry, we really have the opportunity to better understand these subsets of children in terms of their underlying conditions, in terms of their ages at presentation, in terms of the types of medications that they are initiated on, and medications that are either added or withdrawn in ways that we really never could do before.

I think that there are a couple of things that we really can look forward to in the pediatric pulmonary hypertension community. There is a very, very recent paper that is just now in press looking at how the genetic influences in pediatric pulmonary hypertension differ from those in adult pulmonary hypertension. I think that as we look at those different genetic influences in the pediatric population, we are also seeing differences in things like age of onset of the disorder, and who knows as we go forward and collect more data what other kinds of differences we will see. So I think that these are real opportunities for us to think about how to better tailor our treatment and our follow-up.

There may be certain genetic differences, for instance, that respond better to targeting certain pathways. There may be conditions, as I previously mentioned, that really tend to get better over time. They're others that, of course, may not get better over time. So we will have the opportunity to learn more about that in children by better understanding their genetic background and the setting of pulmonary hypertension and pulmonary vascular disease.

I also think we have a great opportunity to really learn a lot more about medications that we use for pulmonary hypertension in children and how do their pharmacokinetics and pharmacodynamics differ from adults. As a neonatologist, I have a particular propensity toward thinking about newborns, and of course, developmentally many things are changing. Also, their metabolism of drugs changes over time. I think as we see these conditions presenting at birth and are really best trying to understand how to approach intervention, learning more about pharmacokinetics and the pharmacodynamics of medications, different medication classes, and different specific medications in those patient populations will be really critical to trying to optimize outcomes.

Dr. Roberta Keller discusses the work of the Pediatric Pulmonary Hypertension Network, (PPHNet) on the network's 10th anniversary. PPHNet is a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease.

My name is Dr. Roberta Keller. I'm a professor of pediatrics at the University of California San Francisco Benioff Children's Hospital here in San Francisco. I'm a neonatologist, and my primary clinical and research interests is in the evaluation and management of newborns and children with cardiopulmonary disorders.

Today, I'd like to talk to you a little bit about the Pediatric Pulmonary Hypertension Network, or PPHNet.

We are a group of ten centers now, which was originally formed about 10 years ago. We're just celebrating our 10th anniversary. It was really the brainchild of Dr. Steve Abman at the University of Colorado and Colorado Children's Hospital in Denver. He saw a real need to bring together people who took care of children with pulmonary vascular disease and pulmonary hypertension and really tried to combine our experience and our efforts toward learning as much as possible about evaluating and treating children with pulmonary vascular disease and pulmonary hypertension.

So PPHNet originally started with eight centers. UCSF was one of those original centers. One of the things that's really great about PPHNet is that it represents people who care for these children from all different disciplines. Of course, there are pediatric cardiologists, which have been the mainstay of caring for children with pulmonary vascular disease, but also pediatric pulmonologists, pediatric critical care physicians, and neonatologists and really all working together toward the common goal of improving our understanding and treatment of affected children.

One of the great things about that is that we all come from different perspectives and have different ways of thinking about the problems that the children present. Of course, one of the challenges is that we aren't always going to agree on how to approach things or what to focus on specifically. We have some very, very lively discussions around how to focus our efforts. I think really it's been very, very productive because it makes us think about things in a different way, consider other perspectives, and really try to bring all of those view together toward improving the care of these patients through our work.

I think one of the things that's really unique about pediatrics is that we really consider growth and development in all of the decisions we make about children in terms of the state of their disease or condition and whether they could potentially benefit from additional therapies or whether we can potentially start to withdraw therapies over time.

In the adult pulmonary hypertension community and adult cardiovascular community, functional class has always been an important way of thinking about how people function in the setting of their disorder or their condition. If you look at the traditional or standard functional class assessments, those are pretty difficult to apply to certainly younger children, as they have a lot to with people's activities of daily living and when they become short of breath.

Honestly, in children, sometimes that's not even possible to get that history from somebody, whether or not they're short of breath. Their activities of daily living are very different over time. We think about a six-month-old versus a one-year-old versus a three-year-old, that is a constantly changing target. We really like to think about functional class in a way that is more specific to children who are growing and developing and incorporating both developmental milestones as well as nutrition and growth and what's needed for them to have adequate nutrition, and of course finally, whether or not they're able to stay in school and how they function in that environment.

The Pulmonary Vascular Research Institute, or PVRI, several years ago in 2011 published a proposed pediatric functional class, which has really helped us with thinking about these children in terms of their status over time and whether they are improving with their therapies or whether they're stalling out or actually worsening. That's been incredibly helpful.

I think that there's other ways of doing this without formally assigning a pediatric functional class. Those are of course are important, but it really incorporates the things we think about, for instance, related to growth. Are children growing well? Are they not growing well? Somebody who might seem to have stable disease, or even mildly improving disease, by imaging or by cardiac catheterization, may in fact not be growing well. So we decide not to withdraw therapies that we might otherwise withdraw in somebody who had those improving metrics. Similarly, a child who seems to have stable numbers but who is falling off in their growth or development or missing more school, we might decide to escalate therapies in order to try and improve their functional status.

One of the things that we worked really hard on, and I think has been covered before in this podcast, are the guidelines of pediatric pulmonary hypertension. Those guidelines are really a slimmed down version of all the work that we did to try and evaluate all the different aspects of pulmonary vascular disease and the evaluation and management of children with that condition.

I think if you look at the literature now, we see that many people are citing those guidelines, many people are reading those guidelines, and they're really contributing to initiation of evaluation of patients prior to them being referred to comprehensive centers and I hope really contributing to the understanding that many practitioners have of the pulmonary vascular disease and how it contributes to the conditions that the children may have as a lot of pulmonary vascular disease and pulmonary hypertension in pediatrics is actually secondary to other conditions, which is unique I think from the adult population.

We've also looked at a number of these conditions more in-depth and published some papers to help the overall community guide their initial approach to those kinds of patients and also identify what the gaps in knowledge are and what we need to really think about going forward.

Now, in PPHNet, we have been working on a registry. We have almost 1,500 children with pulmonary vascular disease in that registry consented for their information to be included. We're now able to look at those data, of course without any identifying identifiers, but in aggregate, to try and understand how these conditions are presenting variably in different children at different ages and what types of therapies we are using and whether or not we can tell if those therapies are efficacious.

In addition, I think what we see in children in general is that many conditions can actually improve over time with organ growth or development. I think we may be seeing that in the pediatric population that we don't just see the clinical improvement over time or functional improvement over time, but maybe sometimes even resolution because those children are growing new lung tissue and new vascular tissue and really able to improve vastly clinically and come off of their medications.

Now, whether those will be long-term successes or whether they will need to at some point go back on medications, we don't know yet. Obviously, we're all anxious about that and continuing to follow those children very closely. But I think that now with that large registry, we really have the opportunity to better understand these subsets of children in terms of their underlying conditions, in terms of their ages at presentation, in terms of the types of medications that they are initiated on, and medications that are either added or withdrawn in ways that we really never could do before.

I think that there are a couple of things that we really can look forward to in the pediatric pulmonary hypertension community. There is a very, very recent paper that is just now in press looking at how the genetic influences in pediatric pulmonary hypertension differ from those in adult pulmonary hypertension. I think that as we look at those different genetic influences in the pediatric population, we are also seeing differences in things like age of onset of the disorder, and who knows as we go forward and collect more data what other kinds of differences we will see. So I think that these are real opportunities for us to think about how to better tailor our treatment and our follow-up.

There may be certain genetic differences, for instance, that respond better to targeting certain pathways. There may be conditions, as I previously mentioned, that really tend to get better over time. They're others that, of course, may not get better over time. So we will have the opportunity to learn more about that in children by better understanding their genetic background and the setting of pulmonary hypertension and pulmonary vascular disease.

I also think we have a great opportunity to really learn a lot more about medications that we use for pulmonary hypertension in children and how do their pharmacokinetics and pharmacodynamics differ from adults. As a neonatologist, I have a particular propensity toward thinking about newborns, and of course, developmentally many things are changing. Also, their metabolism of drugs changes over time. I think as we see these conditions presenting at birth and are really best trying to understand how to approach intervention, learning more about pharmacokinetics and the pharmacodynamics of medications, different medication classes, and different specific medications in those patient populations will be really critical to trying to optimize outcomes.

My name is Dr. Roberta Keller, and I'm aware that I'm rare. Learn more about pulmonary hypertension atwww.phaware365.global. Never miss an episode with the phaware® podcast app. Follow us @phaware on Facebook, Twitter, Instagram, YouTube & Linkedin Engage for a cure: www.phaware.global/donate #phaware #phawareMD @PPHNet @UCSFChildrens

]]>Dr. Roberta Keller discusses the work of the Pediatric Pulmonary Hypertension Network, (PPHNet) on the networks 10th anniversary. PPHNet is a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease.

]]>10:11cleannovel,blood,research,heart,pediatric,awareness,therapies,asthma,breathless,rare,ph,pulmonary,raredisease,catheterization,pulmonaryhypertension,phaware,lungdiseaseDr. Roberta Keller discusses the work of the Pediatric Pulmonary Hypertension Network, (PPHNet) on the network's 10th anniversary. PPHNet is a network of clinical specialists, researchers, and centers bringing a collaborative and multidisciplinary approach to improving care for children with pulmonary vascular disease.
Learn more about pulmonary hypertension at www.phaware365.global. Follow us @phaware Engage for a cure: www.phaware.global/donate #phaware #phawareMD280fullphaware global associationAlvin Rocha, RN - phaware® interview 183Fri, 24 Aug 2018 12:00:00 +0000

Alvin Rocha, RN discusses clinic visits in the outpatient setting and his role in the Pediatric Pulmonary Hypertension Program of Children’s Hospital Los Angeles (CHLA).

My name is Alvin Rocha and I'm from Los Angeles, California. I work at Children's Hospital Los Angeles.

I was hired as the new pulmonary hypertension nurse care manager is what we call it, similar to a nurse coordinator position. I'm new to the pulmonary hypertension world. I've been working in it since July of last year and I love it.

I've worked with a couple of [PH] patients. I was a bedside nurse for about five years, and I worked with a couple of pulmonary hypertension patients in the hospital. I just fell in love with that population and I'm interested in finding out new things we can do for the pediatric population. There's a lot of work that needs to be done and I'm totally motivated to do it.

My main role at Children's Hospital is to be part of the clinic visit in the outpatient setting. When patients have their appointments with their cardiologist, I'll be part of that visit. I check in with the patient, I see how they're doing with their symptoms, with their medication management. I see if there's any help they need with insurance issues or anything in general. I kind of act as their main resource in the cardiology department and then help them navigate through any difficulties they're having at home. I can contact their pharmacy for them, I can contact other doctors for them, and I can even walk them through they're feeling at home and see how we can help with that.

Part of the role is just to be essentially part of the patient's family. I'm the first person they call if they have any questions about anything. For me to act as the advocate and fight for the patients makes me feel like I'm part of their family. It takes time to get to know everybody. I'm still meeting all my patients and what I like about it is that they call me about anything. I've had patients call me about things going on at school. They don't have the courage to talk to their parents about certain things, so they talk to me about it and I'll kind of guide them through their problems.

That's what I like about it, is I'm more part of the family, and it takes time to gain the trust of everybody. As the barriers are broken between us, I become someone that they can come to without feeling like they're calling their doctor. They're calling the nurse who's guiding them through this disease.

I've found that a lot of parents do their own research. They go to conferences, they look online, they Google things. What I've also learned, though, is that every PH patient is different. Every kid growing up with a certain medication has a different life. They do different activities. Kids are athletes, kids are bookworms, kids don't like mingling with other people, some kids are all about meeting other people. But the parents are the ones who kind of have the common ground where they are so hopeful all the time and they just feel like they don't really get clear answers, but that's because there's a huge team behind them looking for answers, and they're all different, other centers do different things, so I find that with parents, my thing is to let them know that there's a lot going on. There's a lot of research, there's a lot of hope, but every center does things differently.

I tell them it's okay to be educated but you have to understand — your son, your daughter, they have their own path. It just depends on the situation. I've had patients that have been on IV medications all their lives, they don't know anything. That's all they know is that they have this pump. They don't take it off, and that's kind of how it goes. It doesn't bother her at all, but when I talk to the parents separately about it, it's very hard still to this day, 10 years later it's hard to see this pump even if they manage it every day. More the parents have a hard time, and I totally understand.

I'm really surprised at how much we still have to learn about pediatric pulmonary hypertension. The treatments are so "young," in the PH world for kids and I'm interested to see over time how we integrate adult treatments into pediatrics and how we can change their lives.

It's a great population to work with. There's so much I want to learn still, and I'm taking baby steps because it is a lot to learn and it's very specialized. But there are great doctors out there, there are great nurses and coordinators out there that are there for the patients. Being someone new to the pulmonary hypertension community, I can promise you that these people are dedicated. There's a group of people that I see at the same conference that will do anything for these patients, and I'm one of those people now, I feel like.

Alvin Rocha, RN discusses clinic visits in the outpatient setting and his role in the Pediatric Pulmonary Hypertension Program of Children’s Hospital Los Angeles (CHLA).

My name is Alvin Rocha and I'm from Los Angeles, California. I work at Children's Hospital Los Angeles.

I was hired as the new pulmonary hypertension nurse care manager is what we call it, similar to a nurse coordinator position. I'm new to the pulmonary hypertension world. I've been working in it since July of last year and I love it.

I've worked with a couple of [PH] patients. I was a bedside nurse for about five years, and I worked with a couple of pulmonary hypertension patients in the hospital. I just fell in love with that population and I'm interested in finding out new things we can do for the pediatric population. There's a lot of work that needs to be done and I'm totally motivated to do it.

My main role at Children's Hospital is to be part of the clinic visit in the outpatient setting. When patients have their appointments with their cardiologist, I'll be part of that visit. I check in with the patient, I see how they're doing with their symptoms, with their medication management. I see if there's any help they need with insurance issues or anything in general. I kind of act as their main resource in the cardiology department and then help them navigate through any difficulties they're having at home. I can contact their pharmacy for them, I can contact other doctors for them, and I can even walk them through they're feeling at home and see how we can help with that.

Part of the role is just to be essentially part of the patient's family. I'm the first person they call if they have any questions about anything. For me to act as the advocate and fight for the patients makes me feel like I'm part of their family. It takes time to get to know everybody. I'm still meeting all my patients and what I like about it is that they call me about anything. I've had patients call me about things going on at school. They don't have the courage to talk to their parents about certain things, so they talk to me about it and I'll kind of guide them through their problems.

That's what I like about it, is I'm more part of the family, and it takes time to gain the trust of everybody. As the barriers are broken between us, I become someone that they can come to without feeling like they're calling their doctor. They're calling the nurse who's guiding them through this disease.

I've found that a lot of parents do their own research. They go to conferences, they look online, they Google things. What I've also learned, though, is that every PH patient is different. Every kid growing up with a certain medication has a different life. They do different activities. Kids are athletes, kids are bookworms, kids don't like mingling with other people, some kids are all about meeting other people. But the parents are the ones who kind of have the common ground where they are so hopeful all the time and they just feel like they don't really get clear answers, but that's because there's a huge team behind them looking for answers, and they're all different, other centers do different things, so I find that with parents, my thing is to let them know that there's a lot going on. There's a lot of research, there's a lot of hope, but every center does things differently.

I tell them it's okay to be educated but you have to understand — your son, your daughter, they have their own path. It just depends on the situation. I've had patients that have been on IV medications all their lives, they don't know anything. That's all they know is that they have this pump. They don't take it off, and that's kind of how it goes. It doesn't bother her at all, but when I talk to the parents separately about it, it's very hard still to this day, 10 years later it's hard to see this pump even if they manage it every day. More the parents have a hard time, and I totally understand.

I'm really surprised at how much we still have to learn about pediatric pulmonary hypertension. The treatments are so "young," in the PH world for kids and I'm interested to see over time how we integrate adult treatments into pediatrics and how we can change their lives.

It's a great population to work with. There's so much I want to learn still, and I'm taking baby steps because it is a lot to learn and it's very specialized. But there are great doctors out there, there are great nurses and coordinators out there that are there for the patients. Being someone new to the pulmonary hypertension community, I can promise you that these people are dedicated. There's a group of people that I see at the same conference that will do anything for these patients, and I'm one of those people now, I feel like.

Anna Brown, RN discusses pulmonary hypertension patients transitioning from pediatrics to adult program as well as the need for comprehensive palliative care that utilizes social workers and psychologists, and mental health providers. Anna is pediatric nurse practitioner at the Vanderbilt Children's Hospital.

My name is Anna Brown. I'm a pediatric nurse practitioner at the Vanderbilt Children's Hospital in Nashville Tennessee. I've been working in the Pediatric Pulmonary Hypertension program there for a little over five years, in the program with Dr. Eric Austin, who's the Director of the program.

I do primarily patient care, whether that's in-patient consults on our patients in the hospital, or following them in the out-patient setting. Seeing them in clinic for well and sick visits, and helping them in the in-between times over the phone.

Patients can be referred to us from outside centers. It's not unusual for a lot of our patients to have been dealing with these symptoms for several years. Seeing various providers outside of our clinic, whether they're living in another state or they were living outside of the Nashville area and seeing a general practitioner or another specialist. So it's not unusual for patients to go several years with symptoms before anybody has a suggestion that they might have pulmonary hypertension. And then they are at that point referred to our center. And we schedule a diagnostic heart catheterization for them to confirm the diagnosis, and then go from there as far as what our treatment plan is.

It's tough information to swallow when you are given a diagnosis that your child has a terminal illness. And that it's a disease that's going to progress and that at this time we don't have a cure for it. I think giving them the assurance that we're going to be with them every step of the way, as we kind of unfold in their story a little bit more and determine what is causing it and what treatment's going to look like and what changes that's going to have on their daily life.

I like to try, and include them from a very young age, even if it's just tell me the name of your medications, or how many pills do you take? Or, what do you tell your friends at school about this pump that you carry? Just to give them a chance to vocalize things in their own words from a young age. As they get older, in their early teens, then it really does become more of a okay, this isn't just to have conversation, but I need to know that you know how to describe your medications and why you take them. The hope is, by the time they get to age 18 or at some point through college, or after they graduate high school that when they transition to the adult period, it won't be as much of a shock.

We've heard from families that despite our best efforts to prepare, it's still very different. To go over into a center where the families are, the parents especially, are not equally as part (not that they're not equally as part of the discussion), but it really is about the patient. And care is directed, and conversation directed, and phone calls are directed towards that patient, and not so much inclusive with the parents as the primary point of contact.

It's interesting because I think people think of transition as that moment when you go from needing a pediatric center to an adult center. It's more of a process and a span of time and not a moment. And I think some of those transition pains and growing pains, we actually feel in a pediatric office in attempts to prepare the family. So even though, I think it's hard if a patient goes to college and whether or not they have a local PH team or a closer PH team that's working with them, parents are going to call us with concerns. And it's hard sometimes to really help that dynamic change to where it's the patient who is calling.

And honestly, I mean, that's probably true of other areas, not just pulmonary hypertension. If a patient has a toothache, they're going to call their parents, and their parents are going to be the ones that navigate the system to get them in with the dentist. So it's a little bit more pronounced because they have more needs I guess, on the end of medications and health care and activity restrictions and what they can do with pulmonary hypertension. But it really is just a stage of life that's being manifested in many other facets.

Overall, I think it's harder for the parents than it is for the patient. I think by the time they get to where they can go to college, they know the names of their medications, they know how much to take, what their doses are. They can manage their infusion therapies independently. I mean, if they couldn't do those things, we wouldn't let them go off to college. But I think it's more from the parent's standpoint, harder to let go a little bit and trust that it's going to go on okay.

We’re certainly not perfect. And I think just in general across the U.S., mental health is an unmet need. And we don't have enough care providers to provide that. I'm a big believer in palliative care because I think that's probably the next closest thing that we have at Vanderbilt. Palliative care is a team of people, and one thing that they do utilize is social workers and psychologists, and mental health providers. What's difficult and the challenge that we haven't been able to bridge yet, is that they come and see us every three or four months and they really need care on a weekly to every two-week basis. Being involved with our palliative care team to help manage stress, and help manage quality of life and the depression and anxiety that comes with having a chronic illness needs a more regular care plan. We've attempted to connect with local resources for these families. Some of them have local resources and some of them don't, but the thing that's tricky with all mental health is that relationship between the patient and that provider has to work. And even in the patients that have resources in their hometowns, it may not be the right fit for them.

I know there's a lot of a move right now towards telehealth and medicine to provide that to families that either don't have the right fit or the right resources. But in the meantime, we have our palliative care team come to clinic with our patients when they come in, just to check in and see what they can do to help. What are the stressors right now on the family? What we've partnered with schools a lot because a lot of schools have school counselors. That doesn't generally work for the older children, but for the elementary school and even middle school children, that's been helpful. It's also been helpful with some of the patient's siblings, as well. That's probably one of the toughest aspects of this I think, because we can do what we can to care for them medically from a biophysical standpoint, but from a mental health and emotional health standpoint, we are definitely not well equipped as a whole.

I think it's one day at a time. I know for a lot of our patients, especially at the beginning of their journey with pulmonary hypertension, we hear a lot that we go home and our child seems perfectly normal. And then we come to the hospital and you're reminded that they're sick. There's some hard feelings and difficulty to come to our clinic visits every two to three months because they're reminded of what is really going on. And then they go home and they get back into their routine, and it's everyday life. And if they're not on an infusion therapy yet, then looking at your young child, you would have no idea if there's anything wrong.

I think when they come and they leave, I think a successful visit is just continuing to build that trust when sometimes they maybe a little bit skeptical of what we're telling them. And then also that when there are bumps in the road, whether it be they get sick with a cold and they really have a hard time breathing or something malfunctions on their pump, or they run out of medication and didn't realize it, and they go a few days without it. Whatever the difficulty may be, helping them feel more comfortable about troubleshooting that on their own and able to problem solve from the home, with our help of course. But helping just to grow and being able to be parents or siblings or friends or the actual patient with pulmonary hypertension.

Anna Brown, RN discusses pulmonary hypertension patients transitioning from pediatrics to adult program as well as the need for comprehensive palliative care that utilizes social workers and psychologists, and mental health providers. Anna is pediatric nurse practitioner at the Vanderbilt Children's Hospital.

My name is Anna Brown. I'm a pediatric nurse practitioner at the Vanderbilt Children's Hospital in Nashville Tennessee. I've been working in the Pediatric Pulmonary Hypertension program there for a little over five years, in the program with Dr. Eric Austin, who's the Director of the program.

I do primarily patient care, whether that's in-patient consults on our patients in the hospital, or following them in the out-patient setting. Seeing them in clinic for well and sick visits, and helping them in the in-between times over the phone.

Patients can be referred to us from outside centers. It's not unusual for a lot of our patients to have been dealing with these symptoms for several years. Seeing various providers outside of our clinic, whether they're living in another state or they were living outside of the Nashville area and seeing a general practitioner or another specialist. So it's not unusual for patients to go several years with symptoms before anybody has a suggestion that they might have pulmonary hypertension. And then they are at that point referred to our center. And we schedule a diagnostic heart catheterization for them to confirm the diagnosis, and then go from there as far as what our treatment plan is.

It's tough information to swallow when you are given a diagnosis that your child has a terminal illness. And that it's a disease that's going to progress and that at this time we don't have a cure for it. I think giving them the assurance that we're going to be with them every step of the way, as we kind of unfold in their story a little bit more and determine what is causing it and what treatment's going to look like and what changes that's going to have on their daily life.

I like to try, and include them from a very young age, even if it's just tell me the name of your medications, or how many pills do you take? Or, what do you tell your friends at school about this pump that you carry? Just to give them a chance to vocalize things in their own words from a young age. As they get older, in their early teens, then it really does become more of a okay, this isn't just to have conversation, but I need to know that you know how to describe your medications and why you take them. The hope is, by the time they get to age 18 or at some point through college, or after they graduate high school that when they transition to the adult period, it won't be as much of a shock.

We've heard from families that despite our best efforts to prepare, it's still very different. To go over into a center where the families are, the parents especially, are not equally as part (not that they're not equally as part of the discussion), but it really is about the patient. And care is directed, and conversation directed, and phone calls are directed towards that patient, and not so much inclusive with the parents as the primary point of contact.

It's interesting because I think people think of transition as that moment when you go from needing a pediatric center to an adult center. It's more of a process and a span of time and not a moment. And I think some of those transition pains and growing pains, we actually feel in a pediatric office in attempts to prepare the family. So even though, I think it's hard if a patient goes to college and whether or not they have a local PH team or a closer PH team that's working with them, parents are going to call us with concerns. And it's hard sometimes to really help that dynamic change to where it's the patient who is calling.

And honestly, I mean, that's probably true of other areas, not just pulmonary hypertension. If a patient has a toothache, they're going to call their parents, and their parents are going to be the ones that navigate the system to get them in with the dentist. So it's a little bit more pronounced because they have more needs I guess, on the end of medications and health care and activity restrictions and what they can do with pulmonary hypertension. But it really is just a stage of life that's being manifested in many other facets.

Overall, I think it's harder for the parents than it is for the patient. I think by the time they get to where they can go to college, they know the names of their medications, they know how much to take, what their doses are. They can manage their infusion therapies independently. I mean, if they couldn't do those things, we wouldn't let them go off to college. But I think it's more from the parent's standpoint, harder to let go a little bit and trust that it's going to go on okay.

We’re certainly not perfect. And I think just in general across the U.S., mental health is an unmet need. And we don't have enough care providers to provide that. I'm a big believer in palliative care because I think that's probably the next closest thing that we have at Vanderbilt. Palliative care is a team of people, and one thing that they do utilize is social workers and psychologists, and mental health providers. What's difficult and the challenge that we haven't been able to bridge yet, is that they come and see us every three or four months and they really need care on a weekly to every two-week basis. Being involved with our palliative care team to help manage stress, and help manage quality of life and the depression and anxiety that comes with having a chronic illness needs a more regular care plan. We've attempted to connect with local resources for these families. Some of them have local resources and some of them don't, but the thing that's tricky with all mental health is that relationship between the patient and that provider has to work. And even in the patients that have resources in their hometowns, it may not be the right fit for them.

I know there's a lot of a move right now towards telehealth and medicine to provide that to families that either don't have the right fit or the right resources. But in the meantime, we have our palliative care team come to clinic with our patients when they come in, just to check in and see what they can do to help. What are the stressors right now on the family? What we've partnered with schools a lot because a lot of schools have school counselors. That doesn't generally work for the older children, but for the elementary school and even middle school children, that's been helpful. It's also been helpful with some of the patient's siblings, as well. That's probably one of the toughest aspects of this I think, because we can do what we can to care for them medically from a biophysical standpoint, but from a mental health and emotional health standpoint, we are definitely not well equipped as a whole.

I think it's one day at a time. I know for a lot of our patients, especially at the beginning of their journey with pulmonary hypertension, we hear a lot that we go home and our child seems perfectly normal. And then we come to the hospital and you're reminded that they're sick. There's some hard feelings and difficulty to come to our clinic visits every two to three months because they're reminded of what is really going on. And then they go home and they get back into their routine, and it's everyday life. And if they're not on an infusion therapy yet, then looking at your young child, you would have no idea if there's anything wrong.

I think when they come and they leave, I think a successful visit is just continuing to build that trust when sometimes they maybe a little bit skeptical of what we're telling them. And then also that when there are bumps in the road, whether it be they get sick with a cold and they really have a hard time breathing or something malfunctions on their pump, or they run out of medication and didn't realize it, and they go a few days without it. Whatever the difficulty may be, helping them feel more comfortable about troubleshooting that on their own and able to problem solve from the home, with our help of course. But helping just to grow and being able to be parents or siblings or friends or the actual patient with pulmonary hypertension.

]]>Anna Brown, RN discusses pulmonary hypertension patients transitioning from pediatrics to adult program as well as the need for comprehensive palliative care that utilizes social workers and psychologists, and mental health providers. Anna is pediatric nurse practitioner at the Vanderbilt Children's Hospital.

I want to talk about the different pathways that we utilize in treating pulmonary arterial hypertension.

As you know, our first big drug back in the '90s was prostanoid. The prostanoid pathway is clearly important. Certain patients maybe don't have enough prostanoid or don't have enough prostanoid synthase to produce prostacyclin to help dilate their blood vessels and their lungs and help prevent remodeling. Prostanoid's a big pathway, and that tends to be a really heavy hitter. When we get someone really sick, we get them on parental prostanoid therapy.

The endothelin receptor pathway's a big one. We still think endothelin is probably the most potent inflammatory mediator in the human body, a natural mediator that really causes vasoconstriction and remodeling. So by having an endothelin receptor antagonist, like we do with bosentan and subsequently ambrisentan and now macitentan, this has been pretty exciting being able to hit two pathways, a prostanoid and then in about 2002 when bosentan was approved, bosentan and subsequently new drugs. Hitting both pathways is important.

Another thing we learned about pathways is hitting two pathways at once might be a good idea. We've done the AMBITION Study now with tadalafil versus ambrisentan, versus both tadalafil and ambrisentan up front. We saw that the results of ambrisentan plus tadalafil up front were better than either drug alone. So clearly hitting two pathways makes sense.

Now, the third pathway is the nitric oxide pathway. We all feel like that's a very potent one. We would probably give inhaled nitric oxide to everyone if we could. It's a little bit inconvenient, so work is being done in that area. But we've got the PD5 inhibitors, sildenafil and tadalafil. And now we've got the soluble guanylate cyclase activator riociguat to use for PAH and CTEPH.

So now we've got three pathways we can use. Should we use all three pathways? We do this in cancer. We treat people upfront. We blast them out of the water. We give them induction therapy, we give them chemo, sometimes radiation. There's a cancer analogy here. I think treating patients aggressively upfront makes sense. That's why the TRITON Study is now underway, which is hitting three pathways all at once. It's hitting the nitric oxide pathway with tadalafil, the ERA pathway with macitentan, and prostanoid pathway with selexipag versus just tadalafil and macitentan alone. So will adding that prostanoid pathway early upfront make a difference? Many of us think it will.

It's probably true that different patients have different pathways that are more important to them than others. Maybe some patients have abnormal endothelin receptors, maybe some have reduced levels of prostacyclin synthase, or an abnormal NO pathway. These are things we need to learn more. Maybe we can tailor our drugs to certain pathways if we learn more about these pathways. Maybe we'll find out a patient can benefit from just two drugs alone, and they'll do just great. So we need a lot more science, a lot more study to learn how important each pathway might be in the individual person.

Now, keep in mind that as I wrap up here that there are other pathways out there, a lot of other exciting areas being studied now. We're specifically looking at inflammation and also the cancer pathway, analogies to PAH. The future is very exciting in PAH.

I want to talk about the different pathways that we utilize in treating pulmonary arterial hypertension.

As you know, our first big drug back in the '90s was prostanoid. The prostanoid pathway is clearly important. Certain patients maybe don't have enough prostanoid or don't have enough prostanoid synthase to produce prostacyclin to help dilate their blood vessels and their lungs and help prevent remodeling. Prostanoid's a big pathway, and that tends to be a really heavy hitter. When we get someone really sick, we get them on parental prostanoid therapy.

The endothelin receptor pathway's a big one. We still think endothelin is probably the most potent inflammatory mediator in the human body, a natural mediator that really causes vasoconstriction and remodeling. So by having an endothelin receptor antagonist, like we do with bosentan and subsequently ambrisentan and now macitentan, this has been pretty exciting being able to hit two pathways, a prostanoid and then in about 2002 when bosentan was approved, bosentan and subsequently new drugs. Hitting both pathways is important.

Another thing we learned about pathways is hitting two pathways at once might be a good idea. We've done the AMBITION Study now with tadalafil versus ambrisentan, versus both tadalafil and ambrisentan up front. We saw that the results of ambrisentan plus tadalafil up front were better than either drug alone. So clearly hitting two pathways makes sense.

Now, the third pathway is the nitric oxide pathway. We all feel like that's a very potent one. We would probably give inhaled nitric oxide to everyone if we could. It's a little bit inconvenient, so work is being done in that area. But we've got the PD5 inhibitors, sildenafil and tadalafil. And now we've got the soluble guanylate cyclase activator riociguat to us